Tag: rare diseases

Hunter Syndrome: New Drug Approval and the Path Forward

April 14, 2026

Hunter Syndrome: New Drug Approval and the Path Forward

Lilly Deals, Merck Acquisition & CAR-T Updates – The Readout LOUD Podcast

April 9, 2026

Exon Science, Biotech VCs, and PhRMA Leadership: The Readout LOUD Podcast

FDA Approves Gene Therapy for Rare LAD-1 Immune Deficiency | Kresladi

March 27, 2026

FDA Approves Gene Therapy for Rare LAD-1 Immune Deficiency | Kresladi

FDA Approves Denali’s Hunter Syndrome Drug Despite Rare Disease Scrutiny

March 25, 2026

FDA Approves Denali’s Hunter Syndrome Drug Despite Rare Disease Scrutiny

Sarepta Stock Jumps on Positive Early Trial Data for Muscular Dystrophy Drugs

March 25, 2026

Sarepta Stock Jumps on Positive Early Trial Data for Muscular Dystrophy Drugs

Biotech Scorecard: Navigating Pharma Criticism & FDA Decisions

March 19, 2026

Biotech Scorecard: Navigating Pharma Criticism & FDA Decisions

FDA Attacks UniQure’s Huntington’s Treatment: Official’s ‘Hot Take’ Podcast Conflict

March 6, 2026

FDA Attacks UniQure’s Huntington’s Treatment: Official’s ‘Hot Take’ Podcast Conflict

Drug Approval Dilemma: Weighing Risk & Benefit in Rare Diseases | STAT+

March 5, 2026

Drug Approval Dilemma: Weighing Risk & Benefit in Rare Diseases | STAT+

UniQure’s Huntington’s Disease Therapy Faces FDA Delay | STAT+ Exclusive

March 2, 2026

UniQure’s Huntington’s Disease Therapy Faces FDA Delay | STAT+ Exclusive

FDA’s New Pathway Offers Hope for Rare Disease Gene Therapies

March 2, 2026

FDA’s New Pathway Offers Hope for Rare Disease Gene Therapies

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