Fierce Biotech

BridgeBio’s muscle weakness data impress ahead of FDA filing

BridgeBio Pharma has taken another step toward the potential approval of its muscle weakness drug candidate BBP-418, ...

US senator investigates FDA over rare-disease drug denials

A key US Republican senator said he’s launched an investigation into the Food and Drug Administration’s recent denials of ...

BridgeBio Pharma eyes first treatment for rare muscle disorder as trial data shows early progress

BridgeBio Pharma Inc. BBIO on Wednesday announced a recent presentation of interim results from its Phase 3 FORTIFY trial for BBP-418, a treatment for limb-girdle muscular dystrophy type 2I/R9.

US FDA puts partial clinical hold on PepGen's muscle disease drug trial

March 4 (Reuters) - PepGen said on Wednesday that the U.S. Food and Drug Administration has placed a partial clinical hold on its mid‑stage trial of a rare muscle disease drug, sending its shares ...

Gene Therapy for Duchenne Muscular Dystrophy: Genethon Confirms Two-Year Efficacy in Patients Treated with Its Drug Candidate GNT0004 at Therapeutic Dose in the First Phase of ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...
Labiotech.eu

Rare Disease Day: is a regulatory overhaul what the therapeutic field needs?

Rare Disease Day is held on the last day of February to raise awareness for rare diseases and address drug development ...
lex18

Family chooses hope over fear in battle against rare genetic muscle disease

RUSSELL COUNTY, Ky. (LEX 18) — A mother's love drives her to pursue experimental treatment as two of her four children battle a rare genetic disease that slowly weakens their muscles. Laura Moore says ...

‘Painful blow’ as HSE told not to cover €26m-€58m cost of miracle drug for kids suffering with rare muscular disease

A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted by families. Last year, The Irish Sun highlighted the plight of ...
Earth.com

New FDA framework could expand treatment access for rare genetic diseases

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...
Yakima Herald-Republic

Grandview student starts nonprofit to bring awareness to rare diseases

GRANDVIEW — Josue Clara's father, Pascual, was diagnosed with spinal and bulbar muscular atrophy, also known as Kennedy's Disease, when he was still in elementary school. The disease is rare, ...
BioSpace

Cashing In on ‘Creative Fundamentals’ To Fund Rare Disease R&D

Rare disease drug developers struggle to survive in a biopharma investment market that prioritizes large patient populations. Initiatives like the Orphan Therapeutics Accelerator are attempting to ...

Cure Rare Disease and LGMD2L Foundation Announce Multi-Year Partnership to Develop Gene Therapy for Anoctamin 5-Related Disease

Cure Rare Disease (CRD), a non-profit biotechnology organization, today announced a landmark partnership with the LGMD2L Foundation. This collaboration is backed by a generous commitment of $7.65 ...