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Spinal Muscular Atrophy
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Spinal Muscular Atrophy trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Spinal Muscular Atrophy trials may be worth asking aboutNorth America
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GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the…
People with cerebral palsy (CP), muscular dystrophy (MD), spina bifida, or spinal cord injury often have muscle weakness, and problems moving their arms and leg…
The purpose of this study is to compare the efficacy and safety of BMS-986365 versus the investigator's choice of therapy in participants with Metastatic Castra…
Background: Androgen effects in humans are usually (but not always) mediated by the androgen receptor which is coded for by the androgen receptor gene (AR gene…
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer. The trial evaluates safety,…
The purpose of the study is to evaluate the safety, tolerability, and efficacy of the two different treatment combinations of tulmimetostat in participants with…
This is a single-session, case-control study that incorporates digital tools for assessing speech and motor function in motor neuron disease. Patients with moto…
This trial will evaluate the use of one versus two DNA vaccines, delivered concurrently with PD-1 blockade using pembrolizumab followed by treatment with pembro…