Limb girdle muscular dystrophy type 2D/R3

Clinical trials for Limb girdle muscular dystrophy type 2D/R3 explained in plain language.

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Not yet recruiting 1 Terminated 1

One-Time gene infusion aims to restore missing muscle protein in children

Cure Not yet recruiting

Researchers are testing a single intravenous infusion of BBM-L101, an experimental gene therapy designed to deliver a working copy of the SGCA gene, in children aged 4 to under 13 with genetically confirmed limb-girdle muscular dystrophy type 2D/R3. The main goal is to check safe…

Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2D/R3

Phase 1 • Sponsor: Peking University First Hospital • Aim: Cure

Last updated Oct 17, 2026 00:00 UTC

Limb girdle muscular dystrophy type 2D/R3

Get alerted when new Limb girdle muscular dystrophy type 2D/R3 trials appear

One-Time gene infusion aims to restore missing muscle protein in children