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Mayo Clinic reports early results of bespoke gene therapy for rare ALS
Original: Mayo Clinic researchers report early results from therapy designed for a single patient with rare genetic form of ALS
Mayo Clinic researchers have reported early results from a therapy designed for a single patient with a rare genetic form of ALS, or motor neurone disease. The findings, shared via hospital and medical news outlets, point to the growing field of individually tailored treatments for ultra-rare genetic conditions. Details of the patient's outcome and safety data remain limited in initial coverage.
Why now: Personalised therapies for rare diseases are a fast-moving area of medicine, and a treatment made for just one patient is notable.
Evidence
- Mayo Clinic researchers report early results from therapy designed for a single patient with rare genetic form of ALS · South Florida Hospital News
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