Question

Care este rata de succes a terapiei cu celule stem pentru Atrofie Musculară Spinală?

SMA stem-cell 'success' claims are scarce due to minimal data. The few case series describe 'stabilisation' in individual children; no controlled trial has set a consensus rate. Approved therapies (nusinersen, gene therapy) show proven motor and survival benefit in controlled trials; stem cells are not proven to match them. Any benefit, if real, would be slowing of decline — evidence is insufficient for strong claims.

Ce arată dovezile pentru Atrofie Musculară Spinală

Five registered trials and 1 recruiting study address stem-cell therapy in SMA — the smallest landscape among the conditions here. Published data are sparse and largely single-centre observational. Some describe stabilisation or slowed decline over 6–12 months; others minimal change. SMA's rapid natural progression makes any stability potentially notable, but separating stem-cell effects from approved disease-modifying therapies (nusinersen, gene therapy) is difficult without controls. No trial has reversed motor-neuron loss.

Sunt un candidat? → · Spinal Muscular Atrophy: full overview → · Cost Atrofie Musculară Spinală → · Cost →

Revizuit medical de echipa editorială a StemCellAtlas cu Kiian Nadiia, MD, PhD (Paediatric Neurologist · Medical Director, CSM Clinic Network · 12+ yrs in Autism Spectrum Disorders) ale clinicii partenere Stem Plus (Sofia), în funcție de orientările ISSCR, FDA și EMA. Educational information, not medical advice; figures indicative.

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