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The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

Latest News

Tirzepatide Could Replace Daily Hormone Shots for Rare Genetic Fat Disorder | Image Credit: Rutgers University
Tirzepatide Could Replace Daily Hormone Shots for Rare Genetic Fat Disorder

February 2nd 2025

Once-weekly tirzepatide may be an alternative to daily hormone shots in patients with congenital generalized lipodystrophy.

FDA Grants Regenerative Medicine Advanced Therapy to Laru-Zova for XLRP | Image Credit: US Food and Drug Administration
FDA Grants Regenerative Medicine Advanced Therapy to Laru-Zova for XLRP

January 31st 2025

Lerodalcibep Matches Evolocumab in LDL-C Reduction for HoFH | Image Credit: TG Forum
Lerodalcibep Matches Evolocumab in LDL-C Reduction for HoFH

January 29th 2025

FDA Approves Axatilimab 9 mg and 22 mg Vial Sizes for Chronic GVHD | Image Credit: US Food and Drug Administration
FDA Approves Axatilimab 9 mg and 22 mg Vial Sizes for Chronic GVHD

January 16th 2025

Oral Linsitinib Reduces Proptosis in Thyroid Eye Disease in Phase 2b/3 Trial | Image Credit: Raymond Douglas
Oral Linsitinib Reduces Proptosis in Thyroid Eye Disease in Phase 2b/3 Trial

January 15th 2025

Latest CME Events & Activities

Clinical ShowCase™: Forming a Personalized Treatment Plan for a Patient With ANCA-Associated Vasculitis

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