Success in muscular dystrophy is measured heterogeneously across trials, with 'success' often defined as slowing decline rather than reversing damage. Published data suggest approximately 30–50% of participants show measurable stabilisation of muscle strength or reduced rate of deterioration over 12–24 months, though many show no significant change. Outcome depends on disease stage at treatment, dystrophy subtype, and cell dose. Late-stage, severely weakened musculature shows less response than early-intervention cases. Genetic subtype plays a critical role — Duchenne dystrophy trials report lower functional gains than some limb-girdle variants.
Clinical trials in muscular dystrophy have predominantly featured small cohorts and observational designs. Four recruiting studies suggest ongoing clinical investigation, though definitive efficacy data remain limited. Some trials reported modest improvements in muscle strength measures and reduced serum creatine kinase levels following MSC infusion, whilst others showed minimal functional benefit. The heterogeneity of dystrophy subtypes means outcomes differ markedly between Duchenne, Becker, and limb-girdle forms. Current evidence supports MSCs as a potential supportive therapy rather than curative, with strongest signals emerging from early-intervention cohorts. Long-term safety data are sparse, and disease progression often continues despite treatment.
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Medically reviewed by StemCellAtlas’s editorial team with Kiian Nadiia, MD, PhD (Paediatric Neurologist · Medical Director, CSM Clinic Network · 12+ yrs in Autism Spectrum Disorders) of partner clinic Stem Plus (Sofia), against ISSCR, FDA & EMA guidance. Educational information, not medical advice; figures indicative.
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