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Rare Disease

Latest News

Rarediseaseawareness | Image Credit: © Penwin-stock.adobe.com
Is Rare Disease Policy a Stress Test of US Health Care?

February 3rd 2026

The Orphan Drug Act revolutionized rare disease treatment, yet rising costs and access issues challenge equitable health care solutions.

FDA approval. | Image credit: aznan – stock.adobe.com
FDA Approves Gene Therapy Onasemnogene Abeparvovec for Broader SMA Population

November 25th 2025

The approval of selumetinib for adults with neurofibromatosis type 1 adds previous approvals in pediatric patients. | Image credit: BHM - stock.adobe.com
FDA Approves Selumetinib for Adults With Neurofibromatosis Type 1

November 20th 2025

Nobel prize | Image Credit: Jean-Luc Flémal - BE - stock.adobe.com
2025 Nobel Prize in Medicine Honors Discoveries Behind Regulatory T Cells

October 6th 2025

David Mitchell, PharmD, MBA, senior pharmacist manager, assistant clinical professor, UC Davis
How Health Systems, Industry Partnerships Advance Rare Disease Therapies: David Mitchell, PharmD, MBA

September 28th 2025

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