Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating ...
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REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss (Revised)
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study.
This article outlines common CMC problems that are obstacles that steal momentum and create costly detours in the development ...
Using fruit flies, University of Wisconsin–Madison researchers have developed a new model for investigating the genetic ...
In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...
Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in ...
LONDON, GREATER LONDON, UNITED KINGDOM, October 15, 2025 /EINPresswire.com/ -- "Get 20% Off All Global Market Reports With Code ONLINE20 – Stay Ahead Of Trade Shifts, Macroeconomic Trends, And ...
FDA accepts Rocket Pharmaceuticals' resubmission for Kresladi gene therapy in severe LAD-I, with a decision expected by March ...
In a small pivotal trial of an investigational gene therapy, most cases of congenital profound deafness from genetic variants ...
Reqorsa® Gene Therapy is a Potential Treatment for ALK-EML4 Positive Translocated Non-Small Cell Lung Cancer AUSTIN, Texas, ...
Affinia Therapeutics ("Affinia"), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class ...
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