Tag: Rare diseases

June 18, 2026

FDA Approval Path Opens for Breakthrough Huntington’s Disease Gene Therapy – New Hope for Patients

June 11, 2026

Blood Cancer United Acquires Luvelta for Pediatric Compassionate-Use Program

May 7, 2026

Ireland Approves First-Ever EB Gel Treatment for Rare, Painful Skin Condition

February 14, 2026

Weight Loss Drugs & Scurvy Risk: What to Know About Vitamin Deficiencies

February 11, 2026

Rare Disease Trials: Why Access to Hope Remains Limited for Many Families

February 9, 2026

FDA Rejects Regenxbio’s Gene Therapy for Hunter Syndrome

January 11, 2026

FDA Delays Expedited Filing for Dravet’s Drug in Stoke Case

January 6, 2026

Bangladesh Disease Bats Mystery

December 30, 2025

EPP Drug Access Concerns: Patients with Sunlight Pain Worry

December 8, 2025

Gene Therapy Improves Mobility in Spinal Muscular Atrophy Children

November 3, 2025

FDA Submission for Uniqure Huntington’s Drug Questioned

September 23, 2025

Rare Disease Care: Advancements and Innovations

July 21, 2025

Sarepta Duchenne Therapy Crisis: What It Means

July 19, 2025

Sarepta Therapeutics Duchenne Crisis Impact

July 19, 2025

Duchenne Muscular Dystrophy: Gene Therapy Deaths & Future Progress

July 18, 2025

Readout LOUD: FDA Drug Rejection, Sarepta Layoffs