A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulant Patients With Spinal Muscular Atrophy
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein
throughout the body. This means fewer motor neurons - nerve cells that pass impulses from
nerves to muscles to cause movement - are lost, which may improve how well muscles work in
people with SMA. RO7204239 is an investigational anti-myostatin antibody that is designed to
target myostatin. Myostatin plays an important role in the regulation of skeletal muscle size
by controlling growth. Inhibiting myostatin may help muscles grow in size and strength.
RO7204239 in combination with risdiplam, which is designed to increase the amount of SMN
protein throughout the body, has the potential to further improve motor function and clinical
outcomes for people living with SMA.
This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in
children aged 2-10 years with spinal muscular atrophy (SMA) that are ambulant.
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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