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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 custom-designed for a single patient with a rare genetic form of ALS, or motor neurone disease. The treatment, tailored to the patient's specific mutation, marks one of the first examples of personalised medicine applied to the fatal neurodegenerative condition. Details of the patient's response and the programme's next steps remain limited, with the hospital news report offering no further clinical data.
Why now: Personalised therapies for rare fatal diseases are a major emerging story in medicine, and this case offers a rare example for ALS.
Mayo ClinicALSrare genetic ALS
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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