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Rare Disease

Latest News

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

The FDA approved selumetinib for adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas based on the KOMET trial.

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

High cholesterol | Image credit: Anusorn - stock.adobe.com
FDA Expands Evinacumab-dgnb Approval for Children as Young as 1 Year With HoFH

September 26th 2025

David Mitchell, PharmD, MBA
How Health Systems, Pharmacy Teams Tackle Rare Disease Therapy Barriers: David Mitchell, PharmD, MBA

September 24th 2025

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