Northern Minnesota boy is first American to receive groundbreaking treatment for muscular dystrophy
An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.
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Duchenne muscular dystrophy: Five trials to watch
The beginning to the year has been eventful in the Duchenne muscular dystrophy (DMD ... for its stem cell-based therapy deramiocel (CAP-1002). The therapy is somewhat unique in that it consists ...
Patient dies following muscular dystrophy gene therapy, Sarepta reports
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
Capricor Therapeutics Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy
Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease----Data Presented at the 2025 Muscular ...
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Promising Early Results of Investigational Duchenne Muscular Dystrophy Therapy
Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
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Capricor Therapeutics' Cell Therapy for Muscular Dystrophy Patients to Get FDA Priority Review
The Food and Drug Administration has accepted Capricor Therapeutics' application seeking approval of its cell therapy deramiocel for muscular dystrophy patients and granted it a priority review.
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Patient dies following muscular dystrophy gene therapy, Sarepta reports
WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy ... into patient cells. It costs $3.2 million ...
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Duchenne muscular dystrophy: Five trials to watch
Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five DMD trials to keep an eye on in 2025.