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Clinical Trials for Spinal Muscular Atrophies of Childhood

• Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX
• Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Who Are Being Treated With Nusinersen or Risdiplam
• SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4
• Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
• Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
• CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
• A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
• A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
• Pilot Study of Growth Hormon to Treat SMA Typ II and III
• Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
• Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I