Statement from Pam Gavin, President and Chief Executive Officer NORWELL, Mass., October 14, 2026 — The National Organization for Rare Disorders (NORD®) applauds the U.S. Food and Drug Administration (FDA)…
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BioMarin, Jazz Pharmaceuticals, and Otsuka CEOs to Join NORD CEO to Examine What it Takes to Turn Scientific Advancements into Patient Progress WASHINGTON, D.C. and NORWELL, Mass., October 6, 2026…
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Acting FDA Commissioner Kyle Diamantas, NIH Director Jay Bhattacharya, and Director of National Health Communications for the Office of the Surgeon General Stephanie E. Haridopolos to Address 900+ Attendees at…
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The Centers for Medicare & Medicaid Services (CMS) has finalized new Medicaid community engagement (work) requirements that states must begin implementing by January 1, 2027. To help state policy leaders…
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NORD has published a new guide to help people and caregivers living with rare diseases understand expanded access and how to access unapproved medicines. In the guide, NORD answers frequently asked questions including …
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Read moreBy Alison Rockett Frase, Guest Blogger In 1995 our son, Joshua, was born with a very rare muscle disorder called Myotubular Myopathy; an early death was imminent for our first-born. …
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Read moreOn July 15, the Food and Drug Administration released the performance goals letter for the reauthorization of the Prescription Drug User Fee Act, also known as PDUFA VI. The PDUFA …
Continue reading “NORD Issues Statement in Response to PDUFA VI Commitment Letter”
Read moreUPDATE: July 11, 2016 Illinois HB 4576 is close to passing! This bill would establish a Rare Disease Commission that would give patients – many of whom are too young …
Continue reading “Advocacy Alert: Illinois Rare Disease Commission Bill”
Read moreThe following statement was issued by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), as the Senate announced it would not vote on its …
Continue reading “NORD Issues Statement as Senate Postpones Vote on Cures Legislation”
Read moreTime is running out – today may be our last chance before September for anything to happen on the critical Zika package. This Friday, Senators will depart D.C. for their 7-week …
Continue reading “July 13 Day of Action: Last Chance for Zika Funding”
Read moreThe Commissioner of the U.S. Food and Drug Administration, Robert Califf, M.D., will speak at NORD’s annual Rare Diseases & Orphan Products Breakthrough Summit in October. Commissioner Califf will provide …
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Read moreSave the date for Sept. 21-22 for a nationwide day of action! Join NORD and 300+ partner organizations as we ‘rally’ for medical research as part of the annual Rally …
Continue reading “Register: Rally for Medical Research Hill Day 2016, Sept. 21-22”
Read moreA recent article in the Financial Times (June 24, 2016) looked at the growing impact families have on medical research. According to the Center of Strategic Philanthropy at the Milken …
Read moreAs part of its ongoing series to promote physician awareness of rare diseases, the National Organization for Rare Disorders (NORD) has published a Physician Guide to Mitochondrial Myopathies, available free …
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