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FDA Approves First Gene Therapy for Inherited Hearing Loss

The ruling sets a precedent for inner-ear gene delivery that could spur more treatments.

HHS Chief Distances From FDA Replimune Denial as Oncologists Dispute Review

Biogen to Buy Apellis for $5.6 Billion in Cash, Adding Syfovre and Empaveli

SCYNEXIS Acquires Poxel’s PXL-770 for ADPKD, Plans 2026 Phase 2 Trial

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

Japan Announces Genomic Medicine Promotion Organization Launch for March 30

Sarepta Reports Early siRNA Data Showing Muscle Delivery as Shares Surge

FDA Grants Accelerated Approval to Avlayah for Neurologic Manifestations of Hunter Syndrome

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

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

Brazil’s SUS Rolls Out Whole‑Exome Sequencing to Speed Rare‑Disease Diagnosis

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

Andalusia Launches 109-Measure Rare Disease Plan as Fundraising Boosts Genomic Testing

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