gene therapy
-
healthGene Therapy Offers Hope for Friedreich Ataxia Cardiomyopathy
A trial shows gene therapy may alleviate heart damage in Friedreich ataxia, with potential for future treatments.
-
healthFDA Draft Guidance Aims to Speed Up Genome Editing Therapies
The FDA proposes letting companies use existing data for genome therapies, aiming to speed development for rare diseases.
-
healthB.C. Family Walks 120 km for Son's Gene Therapy Funding
The Gill family completed a 120 km walk to raise awareness for their son's rare disease and gene therapy.
-
healthFDA's New Pathway for Gene Therapies: Safety vs. Speed for Rare Diseases
The FDA implements a new framework allowing gene therapies for ultra-rare diseases without traditional clinical trials, sparking debate over safety and efficacy.
-
healthLEO Pharma Acquires Replay's Gene Therapy Platform for $50 Million
LEO Pharma acquires Replay's HSV gene therapy platform for $50M upfront, targeting rare dermatological conditions like dystrophic epidermolysis bullosa.
-
healthFDA Approves Groundbreaking Gene Therapy for Genetic Hearing Loss
The FDA has approved Otarmeni, the first gene therapy to treat severe to profound hearing loss linked to the OTOF gene, offering new hope for patients.
-
healthFDA Approves First Gene Therapy for Inherited Deafness
Regeneron's Otarmeni receives FDA approval, marking a significant advancement in treating OTOF gene-related hearing loss in children and adults.
-
healthGene Therapy Breakthrough Restores Hearing in 90% of Inherited Deafness Patients
Largest trial of its kind shows experimental gene therapy significantly improves hearing in individuals with congenital deafness, offering new hope beyond cochlear implants.