A place for the rare disease community to engage!
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New York State Rare Disease Collaborative
If you have a New York State regulated health plan (commerical, marketplace, or Managed Medicaid), there are new rules for step therapy which take effect January 1, 2026. Step therapy is a cost control practice where patients must try and fail on a less expensive medication before they can receive a more expensive medication. For more information on the new rules, CLICK HERE.
If you have a New York State regulated health plan (commerical, marketplace, or Managed Medicaid), there are new rules for step therapy which take effect January 1, 2026. Step therapy is a cost control practice where patients must try and fail on a less expensive medication before they can receive a more expensive medication. For more information on the new rules, CLICK HERE.
Bill Will Help Children with Rare/Fragile Conditions
Bill Will Help Children with Rare/Fragile Conditions
Bill A.2384/S.965 Enables out-of-network coverage
Bill A.2384/S.965 Enables out-of-network coverage
A new bill from Assemblyman Eichenstein (48/D) and Senator Hoylman-Sigal (47/D), can help children with rare and other serious medical conditions get care by making it easier for their families to seek treatment from out-of-network specialists.
NYS RDAC Bill Returns!
NYS RDAC Bill Returns!
Bill creates a New York State rare disease advisory council
Bill creates a New York State rare disease advisory council
Bill S.1287a / A.1296a, from Senator Roxanne Persaud (19/D) & Assemblywoman Amy Paulin (88/D), passed the full NY Senate unanimously in 2025, and made it as far as the Assembly Ways & Committee before the legislature recessed. In 2026 the bill will return for consideration, and rare disease advocates will be there to push again for passage!
New Mental Health Survey for Rare Disease Patients!
New Mental Health Survey for Rare Disease Patients!
Survey will help study mental health needs for rare patients in US & UK
Survey will help study mental health needs for rare patients in US & UK
A new survey has been released to study the mental health needs of people with rare diseases, and people from the United States and United Kingdom are welcome to try it.
Rare NY Spotlight
Rare NY Spotlight
Dynamic Mother/Daughter Duo shine a light for people with Hermansky Pudlak Syndrome
Dynamic Mother/Daughter Duo shine a light for people with Hermansky Pudlak Syndrome
Meeting Ashley Appell, you can’t miss her unique brand of spunk. It’s a mix of wanting to hug the world with a determination to make it a better place. Then you meet mom Donna and quickly learn where Ashley gets it from, and how this duo became such empowered advocates for people with Hermansky Pudlak Syndrome.
Some facts about rare diseases in the US:
Some facts about rare diseases in the US:
A rare disease is one which affects fewer than 200,000 people in the United States. (Source: US Food & Drug Administration)
There are an estimated 10,000 rare diseases. (Source: US National Institutes of Health)
An estimated 25 - 30 million Americans, or 1 in 10, have a rare disease. (Source: US National Institutes of Health)
Almost half of all people with a rare disease are children. (Source: Every Life foundation)
On average, it take a person an average of 6 years to receive a diagnosis. (Source: National Organization of Rare Diseases)
Almost 95% of rare diseases lack a specific FDA approved treatment. (Source: US Food & Drug Administration)
Find help for managing the life challenges associated with having a rare disease.
Read up on health policy news and issues affecting people with rare diseases in New York State.
Look for organizations that offer programs for people with rare diseases and support research.
A collection of tips and information to help you advocate for your community and your issues.
Learn more about treatment for, and research into, rare diseases from public sources.
News and events from New York State and beyond specifically for people with rare diseases.
There's strength in numbers!
There's strength in numbers!
People with different rare diseases face many of the same challenges, including finding a diagnosis, getting quality health care, and managing the life struggles associated with having a rare disease. By working together to learn, share, and advocate we can make a difference for us all!
Dysautonomia International Launches Fellowship Program
Dysautonomia International Launches Fellowship Program
New Program Will Help Improve Patient Access to Care
New Program Will Help Improve Patient Access to Care
In December the Dysautonomia International association launched the first Dysautonomia International Autonomic Disorders Fellowship, a one-year advanced clinical training program designed to prepare physicians to become experts in autonomic nervous system disorders.
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