Myotubular Myopathy

  • NatHis-CNM: Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies
  • INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger
  • ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients

 

Acronym

NatHis-CNM

INCEPTUS

ASPIRO

Study Type RBM-HPS RBM-HPS RBM
Intervention AT132
Trial type International multicenter trial International multicenter trial International multicenter trial
Principal investigators
Laurent Servais Laurent Servais Laurent Servais
Sponsor AIM Audentes Therapeutics Audentes Therapeutics
Fundings Dynacure / Valerion / Généthon Audentes Therapeutics Audentes Therapeutics
Study status Ongoing Ongoing Ongoing
Recruitment status Ongoing Ongoing Ongoing
Population Adult / Child Child Child
Contact l.servais@institut-myologie.org l.servais@institut-myologie.org l.servais@institut-myologie.org
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