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Friedreich’s Ataxia News highlighted 2024's top FA research and treatments, including vatiquinone's FDA approval pursuit, gene therapy advancements, and novel treatments like DT-216P2 and PPL-001. Studies showed improvements in FA symptoms, mitochondrial function, and heart defects in mice, aiming for better FA management.

The article highlights Voyager Therapeutics, Inc. (NASDAQ:VYGR) among the top gene therapy stocks, emphasizing gene therapy's role in treating diseases by modifying genes. It notes the US gene therapy market's growth to $3,697.8 million by 2033, driven by genetic disorder prevalence, advancements, and FDA support, including 38 approved therapies and trends like personalized manufacturing and ML for data management.

Gene therapy, a medical technique modifying genes to treat diseases, is growing rapidly. The US market, valued at $1,312.2M in 2024, is expected to reach $3,697.8M by 2033. Key growth drivers include genetic disorder prevalence, advancements in genetic engineering, and R&D investments. The article lists 10 top gene therapy stocks to buy, highlighting their potential in treating diseases like sickle cell disease and Alzheimer's.

Eli Lilly's Prevail Therapeutics is evaluating PR001, an AAV9 gene therapy for Parkinson's disease with GBA1 mutations in the PROPEL trial. The open-label, ascending dose study, initiated in 2020, includes one-time PR001 treatment with methylprednisolone and optional sirolimus. The trial, recruiting in the U.S. and Israel, has a 2029 completion date and primary end points include treatment-emergent adverse events and immunogenicity. Additional studies evaluate PR001 for type 2 Gaucher disease.

Dr. Horan, Dr. Sauder, Dr. Ramsay, and Dr. Yohn are Bristol Myers Squibb employees. Dr. Harvey has affiliations with multiple companies, including Bristol Myers Squibb. Dr. Ramsay holds Bristol Myers Squibb stock. Dr. Keefe consults for various companies and has received royalties. Dr. Davis consults for several companies. Dr. Paul has roles at Karuna Therapeutics, Sage Therapeutics, and Voyager Therapeutics. Dr. Brannan was with Karuna Therapeutics and now consults for Bristol Myers Squibb.

JMP Securities initiated coverage on Solid Biosciences with a Market Outperform rating and a $15 price target, citing its leadership in gene therapy for DMD, strong liquidity, and potential in capsid engineering. The firm sees significant upside, with a $204.17M market cap and promising clinical and preclinical assets.

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DelveInsight's report on the Gene and Cell Therapies Targeting CNS Disorders market forecasts growth to 2034, highlighting key companies and therapies, including Bayer AG's Phase Ib clinical trial success for Parkinson's disease and uniQure's positive trends in Huntington's disease trials. The market, valued at approximately USD 1 billion in 2021, is expected to surge due to increasing disease prevalence and pipeline product launches.

Alzheimer’s affects 6M Americans, projected to double by 2050. Early detection is crucial for quality of life, but 33-44M with dementia remain undiagnosed. Innovations like blood tests for pTau217 offer scalable, accessible early diagnosis, paving the way for personalized medicine and improved care outcomes.

Tau Inhibitors Clinical Trial Pipeline Analysis shows 25+ key companies expected to transform treatment, with increased funding accelerating advancements in neurodegenerative disease therapies.