Muscular Atrophy clinical trials at UCLA

2 research studies open to eligible people

Showing trials for

  • NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

    open to eligible people ages 18-75

    The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3

    Los Angeles, California and other locations

  • Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

    open to all eligible people

    Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an…

    Los Angeles, California and other locations

Our lead scientists for Muscular Atrophy research studies include .

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