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FDA Approves First Therapy for Acquired Hypothalamic Obesity, Expanding Rhythm’s Imcivree

The decision follows a late-stage study that showed a 15.8% BMI reduction at 52 weeks compared with a 2.6% increase on placebo.

Rare Disease Day Spurs Calls for Faster Diagnosis and Fair Access to Care

EU Regulators Back First Combined COVID-19 and Flu mRNA Shot for Adults 50 and Over

CHMP Backs Xolremdi for WHIM Syndrome Under Exceptional Circumstances

FDA Proposes ‘Plausible Mechanism’ Pathway for Individualized Rare-Disease Therapies

FDA Rejects Disc Medicine’s Bitopertin for EPP After Questioning Surrogate Endpoint

FDA Rejects Regenxbio’s Hunter Syndrome Gene Therapy Application

Sanofi’s Venglustat Notches Gaucher Type 3 Phase 3 Win, Misses Fabry Pain Endpoint

Streeting Seeks Faster, Universal Newborn SMA Screening in England

High-Cost Cancer and Rare-Disease Drugs Push France’s 2024 Reimbursements to €27.2 Billion

FDA Eases CMC Rules for Cell and Gene Therapies to Speed Development

BioMarin to Acquire Amicus for $4.8 Billion in Cash

HHS Adds Duchenne Muscular Dystrophy and MLD to Newborn Screening Recommendations

Kyverna CAR-T Delivers Positive Phase 2 Topline in Stiff Person Syndrome as Shares Jump

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

Doctor-MPs Urge PM to Lift Rare-Disease Funding Cap as ERT Patients Face Immediate Risk

Capricor’s HOPE-3 Trial Reports Cardiac and Muscle Gains in Duchenne Muscular Dystrophy

AI Model popEVE Delivers Proteome-Wide Severity Scores for Rare-Disease Diagnosis

Vigo Hospital Is Galicia’s Only Accredited Center for Balloon Pulmonary Angioplasty

FDA Approves Koselugo for Adults With NF1-Related Plexiform Neurofibromas