gene therapy
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healthStem Cell Transplant Dominates Sickle Cell Economics Over Gene Therapy
New research shows non-myeloablative haploidentical stem cell transplantation delivers superior clinical value at significantly lower cost than gene therapy for sickle cell disease.
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healthBeyond Biohacking: 5 Longevity Trends from Gene Therapy to Neurofeedback
From DNA-based supplements to single-injection gene therapy, the biohacking frontier is expanding beyond diet and sleep. Here are the key trends.
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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.
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healthFDA Draft Guidance Aims to Speed Up Genome Editing Therapies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.
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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.
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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.
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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.
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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.