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Rare Diseases

Drug Access Lysosomal Storage Disorders Prader-Willi Syndrome Batten Disease Friedreich's Ataxia Cortrophin Hematologic Disorders ANCA-associated Vasculitis FDA Guidance Drug Evaluation Pediatric Treatments Facioscapulohumeral Muscular Dystrophy Immunology Cortrophin Gel Charcot-Marie-Tooth Disease AstraZeneca Portfolio Enzyme Replacement Therapy Heart Conditions Neurological Disorders Myasthenia Gravis

HSE Refers Skyclarys Reimbursement to Rare Diseases Review Committee

The referral pauses a final funding decision while specialists reassess the drug's clinical benefit against its high cost.

Regulators Move to Strip Approval of Tavneos After Trial Misconduct and Liver Safety Signals

Angelini Pharma to Acquire Catalyst Pharmaceuticals for $4.1 Billion in Cash

Neurocrine to Acquire Soleno for $2.9 Billion in All-Cash Tender

Sarepta Reports Early siRNA Data Showing Muscle Delivery as Shares Surge

ANI Pharmaceuticals Posts Record Quarter as Rare-Disease Drugs Power 29.6% Sales Jump

FDA Maps New Paths for Rare-Disease Drug Evidence, Keeps Bar High

BioMarin to Acquire Amicus for $4.8 Billion in Cash

China Approves Sanofi’s Qfitlia for Hemophilia A Prophylaxis and Cablivi for aTTP

AstraZeneca Invests $2 Billion to Expand Maryland Biologics and Build New Clinical Site

Sanofi to Acquire Blueprint Medicines for $9.1 Billion, Boosting Its Rare Immunology Pipeline