Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Biohaven Pharmaceuticals, Inc.
Myostatin is a negative regulator of muscle growth. Blocking myostatin activity has been shown to increase muscle size and function. Taldefgrobep alfa directly blocks myostatin activity and was well tolerated in other clinical studies. In combination with medications that increase the amount of SMN protein in the body, taldefgrobep alfa has the potential to further improve motor function and clinical measures for people living with SMA.
Age
4–21
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
Ambulant or Non-Ambulant
Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial including nusinersen and/or risdiplam and/or a history of onasemnogene abeparvovec
You may not be if
Cannot have previously taken anti-myostatin therapies
Must weigh at least 15kg
Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)
History of Spinal Fusion within 6 months of Screening. MAGEC rod nonsurgical adjustments are allowed during the study
Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
Clareo Health | A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
