RARE DISEASE DAY 2/28/2018

Rare Disease Report

NORD: The Voice of the Community – February 2018

FEBRUARY 20, 2018

News from NORD and its Member Organizations – February 2018

Find Out What’s Happening in Your State for Rare Disease Day
On February 28th, people around the world will observe Rare Disease Day. To find out what’s happening around the U.S. and in your state, visit the national website hosted by NORD. Events are planned in state legislative buildings in more than 30 states to promote awareness of issues. In addition, special events are taking place on many university campuses and in schools and hospitals. Post your event here and read a state-by-state overview.

Join NORD in Rare Disease Day Tweetchat
You are invited to a tweetchat on Rare Disease Day (Feb. 28th) hosted by NORD, The Mighty and Boston Children’s Hospital to discuss “What to do if You or Your Child Receive a Rare Disease Diagnosis.” Use hashtag #RDD18Chat. The chat runs from 1 to 2 pm ET. NORD will be live-tweeting from @RareDiseases. Co-hosts will be @TheMightySite and @BostonChildrens. Special guests will include Marsha Lanes MS, CGC, Genetic Counselor and Medical Editor on the NORD staff; Phillip L. Pearl MD, Director of Epilepsy and Clinical Neurophysiology at Boston Children’s and Ashanthi De Silva, Rare Disease Editor for The Mighty.

NORD to Partner Again with The Hole in the Wall Gang Camp
Applications are available for a rare disease summer family camp in Connecticut on which NORD is partnering again this year with The Hole in the Wall Gang Camp. The camp provides a special opportunity for children and families affected by rare diseases to join together for a weekend of pure fun, free of charge. Family Camp is open to 25 families who are located in the Northeast. It will take place May 31-June 3 in Ashford CT.  Apply here.

Letter to Congress: NORD & Others Oppose “Right to Try”
NORD and 40 other patient organizations and professional societies have sent a letter to the leadership of the U.S. House of Representatives explaining why they oppose “Right to Try” legislation currently being considered by Congress. Signers of the letter include the American Society of Clinical Oncology, Leukemia & Lymphoma Society, American Lung Association and National Health Council. The organizations say they support patients being given access to unapproved therapies but believe the bills under consideration won’t increase patient access.

“The Ataxian” Documentary to be Available to Public for Download
A documentary film featuring the cross-country bike ride of two young men living with Friedreich’s ataxia, “The Ataxian”, will be screened in Washington DC as part of the Rare Disease Day activities. In addition, it will be available to the public beginning Feb. 26. Kyle Bryant and Sean Baumstark and their team’s legendary bike ride across America are featured in the film. Beginning Feb. 26th, the film will be available on iTunes and other download sites.

NORD Advocates for Iowa Co-pay Choice Bill
NORD has been advocating for the Iowa Co-pay Choice Bill (SSB 3004). This legislation would help with rising out-of-pocket costs. Specifically, SSB 3004 gives choice and co-pay predictability to Iowa families by requiring that insurance providers offer a minimum number of plans that provide a traditional co-pay option, as opposed to co-insurance. NORD will continue to advocate for this legislation throughout February.

NORD Submits Comments to the Centers for Medicare and Medicaid Services
NORD has been, and will continue to be, engaged in a number of activities to protect access to quality healthcare coverage for Medicaid beneficiaries. Most recently, NORD has submitted comments to the Centers for Medicare and Medicaid Services (CMS) regarding Kansas’ effort to implement work requirementsla and a lifetime limit in its Medicaid program. Read the comments.

NEWS FROM NORD MEMBER ORGANIZATIONS
Alagille Syndrome Alliance Offers Scholarship for Students With Alagille Syndrome
The ALGSA is again offering the Alaina Kaitlyn Hahn Celebratory Scholarship for students with Alagille syndrome. Applications are being accepted now. The scholarship funds the education of high school graduates or GED credential recipients with ALGS who are enrolled in or entering an accredited U.S. university, college or vocational school and choose to pursue higher education in the human biological, medical or health sciences. Write to alagille[@]alagille[.]org and include AKH Scholarship in the subject line to request application materials.

Abstracts Being Accepted for Cornelia de Lange Syndrome Foundation Symposium
Abstracts are currently being acceptedfor the Cornelia de Lange Syndrome Foundation’s 8th Biennial Scientific and Education Symposium. The meeting will take place June 27-28 in Minneapolis. Attendees include physicians in various clinical disciplines, genetic counselors and research scientists.  The meeting encourages interactions between basic and clinical scientists.

Alpha-1 Foundation Awards John Walsh Translational Research Award
The John W. Walsh Translational Research Award has been granted to Joseph Kaserman MD, instructor of medicine at Boston University School of Medicine and a physician in Pulmonary, Allergy, Sleep and Critical Care Medicine at Boston Medical Center. The award was established in memory of John Walsh, co-founder of the foundation who dedicated his life to seeking a cure for alpha-1 antitrypsin deficiency. More.

Opioid Epidemic Impacts Patients With Sarcoidosis
The Foundation for Sarcoidosis Research has issued a press release about a survey on chronic pain, neuropathy and medications for sarcoidosis patients. The survey provides information related to chronic pain experienced by those living with sarcoidosis and the opioid epidemic. Read the press release.

National Tissue Biobank Established for Coats Disease
The Jack McGovern Coats’ Disease Foundation has established a national tissue biobank at the Wilmer Eye Institute of Johns Hopkins Medicine to facilitate research on the causes of, and most effective treatments for, Coats’ disease. This rare disorder affects the retina and has been diagnosed in children as young as four months. More. 

MDS Foundation Hosts Family/Caregiver Forums
The MDS Foundation hosts forums at locations around the U.S. for newly diagnosed patients, long-term survivors or caregivers affected by myelodysplastic syndromes. Medical experts present information on treatment options and strategies for living with MDS. View the dates and locations.

One-Day Conference Planned on Effects of Necrotizing Enterocolitis on Premature Infants and Families
The Morgan Leary Vaughn Fund Inc. is hosting a one-day regional conference in Cromwell CT on June 11 on reducing the devastating effects of necrotizing enterocolitis on premature infants and their families. Online registration is open now for “Speaking of NEC: Unplugged.”

Osteogenesis Imperfecta Foundation Plans Conference
July 13-15 will be the dates of the biennial conference of the Osteogenesis Imperfecta Foundation. To take place in Baltimore, the program will feature forums with leading experts, practical living advice and research.

Progeria Research Foundation Scientific Workshop Planned
The 9th International Scientific Workshop of the Progeria Research Foundation will take place September 20-22 in Cambridge MA. This event promotes collaboration among basic and clinical scientists toward progress in progeria, cardiovascular disease and aging research. Information and registration.

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Rare Disease Report

NORD: The Voice of the Community – February 2018

FEBRUARY 20, 2018

News from NORD and its Member Organizations – February 2018

Find Out What’s Happening in Your State for Rare Disease Day
On February 28th, people around the world will observe Rare Disease Day. To find out what’s happening around the U.S. and in your state, visit the national website hosted by NORD. Events are planned in state legislative buildings in more than 30 states to promote awareness of issues. In addition, special events are taking place on many university campuses and in schools and hospitals. Post your event here and read a state-by-state overview.

Join NORD in Rare Disease Day Tweetchat
You are invited to a tweetchat on Rare Disease Day (Feb. 28th) hosted by NORD, The Mighty and Boston Children’s Hospital to discuss “What to do if You or Your Child Receive a Rare Disease Diagnosis.” Use hashtag #RDD18Chat. The chat runs from 1 to 2 pm ET. NORD will be live-tweeting from @RareDiseases. Co-hosts will be @TheMightySite and @BostonChildrens. Special guests will include Marsha Lanes MS, CGC, Genetic Counselor and Medical Editor on the NORD staff; Phillip L. Pearl MD, Director of Epilepsy and Clinical Neurophysiology at Boston Children’s and Ashanthi De Silva, Rare Disease Editor for The Mighty.

NORD to Partner Again with The Hole in the Wall Gang Camp
Applications are available for a rare disease summer family camp in Connecticut on which NORD is partnering again this year with The Hole in the Wall Gang Camp. The camp provides a special opportunity for children and families affected by rare diseases to join together for a weekend of pure fun, free of charge. Family Camp is open to 25 families who are located in the Northeast. It will take place May 31-June 3 in Ashford CT.  Apply here.

Letter to Congress: NORD & Others Oppose “Right to Try”
NORD and 40 other patient organizations and professional societies have sent a letter to the leadership of the U.S. House of Representatives explaining why they oppose “Right to Try” legislation currently being considered by Congress. Signers of the letter include the American Society of Clinical Oncology, Leukemia & Lymphoma Society, American Lung Association and National Health Council. The organizations say they support patients being given access to unapproved therapies but believe the bills under consideration won’t increase patient access.

“The Ataxian” Documentary to be Available to Public for Download
A documentary film featuring the cross-country bike ride of two young men living with Friedreich’s ataxia, “The Ataxian”, will be screened in Washington DC as part of the Rare Disease Day activities. In addition, it will be available to the public beginning Feb. 26. Kyle Bryant and Sean Baumstark and their team’s legendary bike ride across America are featured in the film. Beginning Feb. 26th, the film will be available on iTunes and other download sites.

NORD Advocates for Iowa Co-pay Choice Bill
NORD has been advocating for the Iowa Co-pay Choice Bill (SSB 3004). This legislation would help with rising out-of-pocket costs. Specifically, SSB 3004 gives choice and co-pay predictability to Iowa families by requiring that insurance providers offer a minimum number of plans that provide a traditional co-pay option, as opposed to co-insurance. NORD will continue to advocate for this legislation throughout February.

NORD Submits Comments to the Centers for Medicare and Medicaid Services
NORD has been, and will continue to be, engaged in a number of activities to protect access to quality healthcare coverage for Medicaid beneficiaries. Most recently, NORD has submitted comments to the Centers for Medicare and Medicaid Services (CMS) regarding Kansas’ effort to implement work requirementsla and a lifetime limit in its Medicaid program. Read the comments.

NEWS FROM NORD MEMBER ORGANIZATIONS
Alagille Syndrome Alliance Offers Scholarship for Students With Alagille Syndrome
The ALGSA is again offering the Alaina Kaitlyn Hahn Celebratory Scholarship for students with Alagille syndrome. Applications are being accepted now. The scholarship funds the education of high school graduates or GED credential recipients with ALGS who are enrolled in or entering an accredited U.S. university, college or vocational school and choose to pursue higher education in the human biological, medical or health sciences. Write to alagille[@]alagille[.]org and include AKH Scholarship in the subject line to request application materials.

Abstracts Being Accepted for Cornelia de Lange Syndrome Foundation Symposium
Abstracts are currently being accepted for the Cornelia de Lange Syndrome Foundation’s 8th Biennial Scientific and Education Symposium. The meeting will take place June 27-28 in Minneapolis. Attendees include physicians in various clinical disciplines, genetic counselors and research scientists.  The meeting encourages interactions between basic and clinical scientists.

Alpha-1 Foundation Awards John Walsh Translational Research Award
The John W. Walsh Translational Research Award has been granted to Joseph Kaserman MD, instructor of medicine at Boston University School of Medicine and a physician in Pulmonary, Allergy, Sleep and Critical Care Medicine at Boston Medical Center. The award was established in memory of John Walsh, co-founder of the foundation who dedicated his life to seeking a cure for alpha-1 antitrypsin deficiency. More.

Opioid Epidemic Impacts Patients With Sarcoidosis
The Foundation for Sarcoidosis Research has issued a press release about a survey on chronic pain, neuropathy and medications for sarcoidosis patients. The survey provides information related to chronic pain experienced by those living with sarcoidosis and the opioid epidemic. Read the press release.

National Tissue Biobank Established for Coats Disease
The Jack McGovern Coats’ Disease Foundation has established a national tissue biobank at the Wilmer Eye Institute of Johns Hopkins Medicine to facilitate research on the causes of, and most effective treatments for, Coats’ disease. This rare disorder affects the retina and has been diagnosed in children as young as four months. More. 

MDS Foundation Hosts Family/Caregiver Forums
The MDS Foundation hosts forums at locations around the U.S. for newly diagnosed patients, long-term survivors or caregivers affected by myelodysplastic syndromes. Medical experts present information on treatment options and strategies for living with MDS. View the dates and locations.

One-Day Conference Planned on Effects of Necrotizing Enterocolitis on Premature Infants and Families
The Morgan Leary Vaughn Fund Inc. is hosting a one-day regional conference in Cromwell CT on June 11 on reducing the devastating effects of necrotizing enterocolitis on premature infants and their families. Online registration is open now for “Speaking of NEC: Unplugged.”

Osteogenesis Imperfecta Foundation Plans Conference
July 13-15 will be the dates of the biennial conference of the Osteogenesis Imperfecta Foundation. To take place in Baltimore, the program will feature forums with leading experts, practical living advice and research.

Progeria Research Foundation Scientific Workshop Planned
The 9th International Scientific Workshop of the Progeria Research Foundation will take place September 20-22 in Cambridge MA. This event promotes collaboration among basic and clinical scientists toward progress in progeria, cardiovascular disease and aging research. Information and registration.

MOST POPULAR ARTICLES
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NORD: The Voice of the Community – February 2018 FEBRUARY 20, 2018 News from NORD and its Member Organizations – February 2018 Find Out What’s Happening in Your State for Rare Disease Day On February 28th, people around the world will observe Rare Disease Day. To find out what’s happening around the U.S. and in your state, visit the national website hosted by NORD. Events are planned in state legislative buildings in more than 30 states to promote awareness of issues. In addition, special events are taking place on many university campuses and in schools and hospitals. Post your event here and read a state-by-state overview.  Join NORD in Rare Disease Day Tweetchat You are invited to a tweetchat on Rare Disease Day (Feb. 28th) hosted by NORD, The Mighty and Boston Children’s Hospital to discuss “What to do if You or Your Child Receive a Rare Disease Diagnosis.” Use hashtag #RDD18Chat. The chat runs from 1 to 2 pm ET. NORD will be live-tweeting from @RareDiseases. Co-hosts will be @TheMightySite and @BostonChildrens. Special guests will include Marsha Lanes MS, CGC, Genetic Counselor and Medical Editor on the NORD staff; Phillip L. Pearl MD, Director of Epilepsy and Clinical Neurophysiology at Boston Children’s and Ashanthi De Silva, Rare Disease Editor for The Mighty. NORD to Partner Again with The Hole in the Wall Gang Camp Applications are available for a rare disease summer family camp in Connecticut on which NORD is partnering again this year with The Hole in the Wall Gang Camp. The camp provides a special opportunity for children and families affected by rare diseases to join together for a weekend of pure fun, free of charge. Family Camp is open to 25 families who are located in the Northeast. It will take place May 31-June 3 in Ashford CT.  Apply here. Letter to Congress: NORD & Others Oppose “Right to Try” NORD and 40 other patient organizations and professional societies have sent a letter to the leadership of the U.S. House of Representatives explaining why they oppose “Right to Try” legislation currently being considered by Congress. Signers of the letter include the American Society of Clinical Oncology, Leukemia & Lymphoma Society, American Lung Association and National Health Council. The organizations say they support patients being given access to unapproved therapies but believe the bills under consideration won’t increase patient access. “The Ataxian” Documentary to be Available to Public for Download A documentary film featuring the cross-country bike ride of two young men living with Friedreich’s ataxia, “The Ataxian”, will be screened in Washington DC as part of the Rare Disease Day activities. In addition, it will be available to the public beginning Feb. 26. Kyle Bryant and Sean Baumstark and their team’s legendary bike ride across America are featured in the film. Beginning Feb. 26th, the film will be available on iTunes and other download sites. NORD Advocates for Iowa Co-pay Choice Bill NORD has been advocating for the Iowa Co-pay Choice Bill (SSB 3004). This legislation would help with rising out-of-pocket costs. Specifically, SSB 3004 gives choice and co-pay predictability to Iowa families by requiring that insurance providers offer a minimum number of plans that provide a traditional co-pay option, as opposed to co-insurance. NORD will continue to advocate for this legislation throughout February.  NORD Submits Comments to the Centers for Medicare and Medicaid Services NORD has been, and will continue to be, engaged in a number of activities to protect access to quality healthcare coverage for Medicaid beneficiaries. Most recently, NORD has submitted comments to the Centers for Medicare and Medicaid Services (CMS) regarding Kansas’ effort to implement work requirementsla and a lifetime limit in its Medicaid program. Read the comments. NEWS FROM NORD MEMBER ORGANIZATIONS Alagille Syndrome Alliance Offers Scholarship for Students With Alagille Syndrome The ALGSA is again offering the Alaina Kaitlyn Hahn Celebratory Scholarship for students with Alagille syndrome. Applications are being accepted now. The scholarship funds the education of high school graduates or GED credential recipients with ALGS who are enrolled in or entering an accredited U.S. university, college or vocational school and choose to pursue higher education in the human biological, medical or health sciences. Write to alagille[@]alagille[.]org and include AKH Scholarship in the subject line to request application materials. Abstracts Being Accepted for Cornelia de Lange Syndrome Foundation Symposium Abstracts are currently being accepted for the Cornelia de Lange Syndrome Foundation’s 8th Biennial Scientific and Education Symposium. The meeting will take place June 27-28 in Minneapolis. Attendees include physicians in various clinical disciplines, genetic counselors and research scientists.  The meeting encourages interactions between basic and clinical scientists. Alpha-1 Foundation Awards John Walsh Translational Research Award The John W. Walsh Translational Research Award has been granted to Joseph Kaserman MD, instructor of medicine at Boston University School of Medicine and a physician in Pulmonary, Allergy, Sleep and Critical Care Medicine at Boston Medical Center. The award was established in memory of John Walsh, co-founder of the foundation who dedicated his life to seeking a cure for alpha-1 antitrypsin deficiency. More. Opioid Epidemic Impacts Patients With Sarcoidosis The Foundation for Sarcoidosis Research has issued a press release about a survey on chronic pain, neuropathy and medications for sarcoidosis patients. The survey provides information related to chronic pain experienced by those living with sarcoidosis and the opioid epidemic. Read the press release. National Tissue Biobank Established for Coats Disease The Jack McGovern Coats’ Disease Foundation has established a national tissue biobank at the Wilmer Eye Institute of Johns Hopkins Medicine to facilitate research on the causes of, and most effective treatments for, Coats’ disease. This rare disorder affects the retina and has been diagnosed in children as young as four months. More. MDS Foundation Hosts Family/Caregiver Forums The MDS Foundation hosts forums at locations around the U.S. for newly diagnosed patients, long-term survivors or caregivers affected by myelodysplastic syndromes. Medical experts present information on treatment options and strategies for living with MDS. View the dates and locations. One-Day Conference Planned on Effects of Necrotizing Enterocolitis on Premature Infants and Families The Morgan Leary Vaughn Fund Inc. is hosting a one-day regional conference in Cromwell CT on June 11 on reducing the devastating effects of necrotizing enterocolitis on premature infants and their families. Online registration is open now for “Speaking of NEC: Unplugged.” Osteogenesis Imperfecta Foundation Plans Conference July 13-15 will be the dates of the biennial conference of the Osteogenesis Imperfecta Foundation. To take place in Baltimore, the program will feature forums with leading experts, practical living advice and research. Progeria Research Foundation Scientific Workshop Planned The 9th International Scientific Workshop of the Progeria Research Foundation will take place September 20-22 in Cambridge MA. This event promotes collaboration among basic and clinical scientists toward progress in progeria, cardiovascular disease and aging research. Information and registration. MOST POPULAR ARTICLES   Vertex Initiates Phase 3 of Triple Combination for Cystic Fibrosis   Sobi Recieves Positive Opinion for Anakinra in Still’s Disease   IFFGD Will Participate in Rare Disease Day to Support Rare Digestive Disease Patients   Does Michael Phelps Have Marfan Syndrome? Strategic Alliance Partnership Program > Infographics > SPECIALTY > Gaucher Disease Familial Chylomicronemia Syndrome Huntington’s Disease Cardiovascular Oncology Neurology MEDICINE > ADVOCACY > PARTNERS > CONTRIBUTORS > PUBLICATIONS > MJH Associates > AJMC CURE MD Magazine HRA ONCLive OTCGuide Pharmacy Times Specialty Pharmacy Times Targeted Oncology Resources About Advertise Careers Contact Us Terms & Conditions Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved. NORD: The Voice of the Community – February 2018 FEBRUARY 20, 2018 News from NORD and its Member Organizations – February 2018 Find Out What’s Happening in Your State for Rare Disease Day On February 28th, people around the world will observe Rare Disease Day. To find out what’s happening around the U.S. and in your state, visit the national website hosted by NORD. Events are planned in state legislative buildings in more than 30 states to promote awareness of issues. In addition, special events are taking place on many university campuses and in schools and hospitals. Post your event here and read a state-by-state overview.  Join NORD in Rare Disease Day Tweetchat You are invited to a tweetchat on Rare Disease Day (Feb. 28th) hosted by NORD, The Mighty and Boston Children’s Hospital to discuss “What to do if You or Your Child Receive a Rare Disease Diagnosis.” Use hashtag #RDD18Chat. The chat runs from 1 to 2 pm ET. NORD will be live-tweeting from @RareDiseases. Co-hosts will be @TheMightySite and @BostonChildrens. Special guests will include Marsha Lanes MS, CGC, Genetic Counselor and Medical Editor on the NORD staff; Phillip L. Pearl MD, Director of Epilepsy and Clinical Neurophysiology at Boston Children’s and Ashanthi De Silva, Rare Disease Editor for The Mighty. NORD to Partner Again with The Hole in the Wall Gang Camp Applications are available for a rare disease summer family camp in Connecticut on which NORD is partnering again this year with The Hole in the Wall Gang Camp. The camp provides a special opportunity for children and families affected by rare diseases to join together for a weekend of pure fun, free of charge. Family Camp is open to 25 families who are located in the Northeast. It will take place May 31-June 3 in Ashford CT.  Apply here. Letter to Congress: NORD & Others Oppose “Right to Try” NORD and 40 other patient organizations and professional societies have sent a letter to the leadership of the U.S. House of Representatives explaining why they oppose “Right to Try” legislation currently being considered by Congress. Signers of the letter include the American Society of Clinical Oncology, Leukemia & Lymphoma Society, American Lung Association and National Health Council. The organizations say they support patients being given access to unapproved therapies but believe the bills under consideration won’t increase patient access. “The Ataxian” Documentary to be Available to Public for Download A documentary film featuring the cross-country bike ride of two young men living with Friedreich’s ataxia, “The Ataxian”, will be screened in Washington DC as part of the Rare Disease Day activities. In addition, it will be available to the public beginning Feb. 26. Kyle Bryant and Sean Baumstark and their team’s legendary bike ride across America are featured in the film. Beginning Feb. 26th, the film will be available on iTunes and other download sites. NORD Advocates for Iowa Co-pay Choice Bill NORD has been advocating for the Iowa Co-pay Choice Bill (SSB 3004). This legislation would help with rising out-of-pocket costs. Specifically, SSB 3004 gives choice and co-pay predictability to Iowa families by requiring that insurance providers offer a minimum number of plans that provide a traditional co-pay option, as opposed to co-insurance. NORD will continue to advocate for this legislation throughout February.  NORD Submits Comments to the Centers for Medicare and Medicaid Services NORD has been, and will continue to be, engaged in a number of activities to protect access to quality healthcare coverage for Medicaid beneficiaries. Most recently, NORD has submitted comments to the Centers for Medicare and Medicaid Services (CMS) regarding Kansas’ effort to implement work requirementsla and a lifetime limit in its Medicaid program. Read the comments. NEWS FROM NORD MEMBER ORGANIZATIONS Alagille Syndrome Alliance Offers Scholarship for Students With Alagille Syndrome The ALGSA is again offering the Alaina Kaitlyn Hahn Celebratory Scholarship for students with Alagille syndrome. Applications are being accepted now. The scholarship funds the education of high school graduates or GED credential recipients with ALGS who are enrolled in or entering an accredited U.S. university, college or vocational school and choose to pursue higher education in the human biological, medical or health sciences. Write to alagille[@]alagille[.]org and include AKH Scholarship in the subject line to request application materials. Abstracts Being Accepted for Cornelia de Lange Syndrome Foundation Symposium Abstracts are currently being accepted for the Cornelia de Lange Syndrome Foundation’s 8th Biennial Scientific and Education Symposium. The meeting will take place June 27-28 in Minneapolis. Attendees include physicians in various clinical disciplines, genetic counselors and research scientists.  The meeting encourages interactions between basic and clinical scientists. Alpha-1 Foundation Awards John Walsh Translational Research Award The John W. Walsh Translational Research Award has been granted to Joseph Kaserman MD, instructor of medicine at Boston University School of Medicine and a physician in Pulmonary, Allergy, Sleep and Critical Care Medicine at Boston Medical Center. The award was established in memory of John Walsh, co-founder of the foundation who dedicated his life to seeking a cure for alpha-1 antitrypsin deficiency. More. Opioid Epidemic Impacts Patients With Sarcoidosis The Foundation for Sarcoidosis Research has issued a press release about a survey on chronic pain, neuropathy and medications for sarcoidosis patients. The survey provides information related to chronic pain experienced by those living with sarcoidosis and the opioid epidemic. Read the press release. National Tissue Biobank Established for Coats Disease The Jack McGovern Coats’ Disease Foundation has established a national tissue biobank at the Wilmer Eye Institute of Johns Hopkins Medicine to facilitate research on the causes of, and most effective treatments for, Coats’ disease. This rare disorder affects the retina and has been diagnosed in children as young as four months. More. MDS Foundation Hosts Family/Caregiver Forums The MDS Foundation hosts forums at locations around the U.S. for newly diagnosed patients, long-term survivors or caregivers affected by myelodysplastic syndromes. Medical experts present information on treatment options and strategies for living with MDS. View the dates and locations. One-Day Conference Planned on Effects of Necrotizing Enterocolitis on Premature Infants and Families The Morgan Leary Vaughn Fund Inc. is hosting a one-day regional conference in Cromwell CT on June 11 on reducing the devastating effects of necrotizing enterocolitis on premature infants and their families. Online registration is open now for “Speaking of NEC: Unplugged.” Osteogenesis Imperfecta Foundation Plans Conference July 13-15 will be the dates of the biennial conference of the Osteogenesis Imperfecta Foundation. To take place in Baltimore, the program will feature forums with leading experts, practical living advice and research. Progeria Research Foundation Scientific Workshop Planned The 9th International Scientific Workshop of the Progeria Research Foundation will take place September 20-22 in Cambridge MA. This event promotes collaboration among basic and clinical scientists toward progress in progeria, cardiovascular disease and aging research. Information and registration. MOST POPULAR ARTICLES   Vertex Initiates Phase 3 of Triple Combination for Cystic Fibrosis   Sobi Recieves Positive Opinion for Anakinra in Still’s Disease   IFFGD Will Participate in Rare Disease Day to Support Rare Digestive Disease Patients   Does Michael Phelps Have Marfan Syndrome? Strategic Alliance Partnership Program > Infographics > SPECIALTY > Gaucher Disease Familial Chylomicronemia Syndrome Huntington’s Disease Cardiovascular Oncology Neurology MEDICINE > ADVOCACY > PARTNERS > CONTRIBUTORS > PUBLICATIONS > MJH Associates > AJMC CURE MD Magazine HRA ONCLive OTCGuide Pharmacy Times Specialty Pharmacy Times Targeted Oncology Resources About Advertise Careers Contact Us Terms & Conditions Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved. ShareThis Copy and Paste

This explains me to a “T”

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We must stop Paul Ryan’s plan to end Obamacare

Please take a minutFB_20160228_15_43_29_Saved_Picturee out of your day and call your State Senators and Congressman/Congresswomen to vote against Paul Ryan. We cannot go back to less than optimal care for our chronic illnesses. http://mobile.reuters.com/article/idUSKCN0XP00C

How do I continue to live with this pain?

Sarcoidosis

Sarcoidosis

I am so tired of this pain. I am so tired of being tired. To describe this pain I would say it’s in my joints, my muscles and tissues and my bones. I take the prescribed pain medication but it doesn’t work. Having trouble sleeping because of the pain. I brought it up to my Sarcoidosis doctor who I now found out is leaving that practice. Don’t know what to do and I hate starting anew with another doctor. In the 16 yrs. since my diagnosis, this would be my fifth pulmonologist. Anywho, just needed to vent and ask for prayer.

Sarcoidosis Research Is Necessary

A friend I never knew Article

http://writeonfighton.org/2016/01/28/a-friend-i-never-knew/

Sarcoidosis Awareness Month is quickly approaching…

MFFL T-ShirtWell that time is quickly approaching again. I am having to recruit much needed help this year with the planning of the Marilyn’s Fight for Life KICK Sarcoidosis Awareness event in April. Thankfully I have those that are stepping up to the plate to help. In my weaking state I have been told to just concentrate on becoming healthy, step back and trust in God to provide what we need, and most importantly NOT 2 STRESS! Everyone that knows me know that I am my fathers child. My mind is thinking a mile a minute. But I digress. I will relax and Let Go/ Let God. I will update soon. Until then, peace and blessings.

Hello Again…

FB_20150526_14_47_33_Saved_PictureHello again. Yesterday I received an infusion that took 5 hrs. Afterwards I was so very tired and worn out. My stomach ached so much to a point of nausea. But today is a new day and I am alive. What you have to understand is that with disease we could drop at the drop of a dime. The heart is the hardest organ to diagnose. It is usually found during autopsy unfortunately. Biopsies are out of the questions even though the symptoms are there. Chest pain, shortness of breath, arrythmias and tireness. This is a very serious disease that needs to be taken seriously. Til next time, keep me in your prayers as I will continue to pray for you.