Rare Disease News

Volunteer Spotlight: How Judy Advocated for Better Access to Care for Rare Hawaii Residents

Published August 19, 2023 by NORD

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This year, NORD Advocate Judy A. testified before the Hawaii state legislature, leading Hawaii to pass a law making it easier for residents with rare diseases to access out-of-state physicians …

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Delaware Enacts Law to Address Needs of Rare Disease Patients and Families

Published July 26, 2023 by NORD

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Delaware is the 27th state to establish a Rare Disease Advisory Council to raise awareness and inform state health policies. July 26, 2023, Dover, DE – Today, patients and families in …

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Final Inflation Reduction Act Guidance Risks Stifling Innovation for Rare Disease Community

Published June 30, 2023 by NORD

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Today, the Centers for Medicare and Medicaid Services (CMS) finalized a key component of the Inflation Reduction Act’s Medicare Drug Price Negotiation Program (MDPNP). Unfortunately, today’s guidance threatens vital innovation …

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National Organization for Rare Disorders Urges Congress To Protect Funding for Rare Disease Research, Commends Exclusion of Work Requirements for Medicaid in Debt Ceiling Deal

Published June 2, 2023 by NORD

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Washington, DC, June 2, 2023 – This week, the Senate passed a debt ceiling deal (HR 3746) by a 63-36 vote and which now goes to President Biden for his …

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Maryland Governor Signs Legislation to Address Needs of Rare Disease Patients and Families Throughout the State

Published May 3, 2023 by NORD

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More than Half of U.S. States Now Solicit Input of State Rare Disease Advisory Councils to Inform Local Health Policies May 3, 2023, Annapolis, MD – Today, patients and families in …

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Congress should protect the intent of the Orphan Drug Act and pass the RARE Act

Published April 24, 2023 by NORD

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Advocacy

Rare disease patients, including children, deserve safe and effective therapies. By Karin Hoelzer, Director of Policy and Regulatory Affairs at NORD This year marks the 40th anniversary of the Orphan …

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Rare Disease Patients and Families Tell CMS to Implement the Inflation Reduction Act in a Way that Works for Our Community

Published April 13, 2023 by NORD

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Advocacy

The Centers for Medicare & Medicaid Services (CMS) is looking to hear from our community about how the Inflation Reduction Act (IRA) should work. The IRA was signed into law …

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Why the Mifepristone Ruling Threatens All FDA-Approved Drugs

Published April 11, 2023 by NORD

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Advocacy

UPDATE: April 24, 2023 — 33 Patient and Provider Groups Applaud Supreme Court Ruling, Urge Courts to Preserve FDA’s Authority Our 33 organizations, representing patients with serious health conditions, applaud …

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NORD’s State Report Card Shows Progress for Rare Disease Patients, Highlights Remaining Barriers

Published February 7, 2023 by NORD

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Patient access to care and services improved across key policy areas in 2022, but challenges remain for the rare disease community February 7, 2023, Washington, D.C. – The National …

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Giving Rare Diseases a Voice: 5 ways to make your voice as a patient advocate heard in 2023

Published January 31, 2023 by NORD

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By Karin Hoelzer Rare disease patients and caregivers often ask me whether they should really engage in health policy. Is it really worth the trouble? In short, the answer is …

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