Study the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067

Clinical Trial

Offered by: Nemours Children's
Location: Orlando

Trial Name

A Two Part Seamless, Open-Label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy.

What is the trial about?

The purpose of this study is to find out if this new drug can improve motor function and stability for children with SMA.

Who can participate?

Children 1–7 months old with SMA Type 1.

What is involved?

48 visits to Nemours Children’s Hospital, Florida over 4 years. Procedures and tests to be done include physical exam, ECG, blood and urine samples, various muscle tests and questions about your medical history.