Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
Status:
Unknown status
Trial end date:
2017-07-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy
on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).