Conference Coverage

What’s New in Rare Neuromuscular Disorders?


 

Debra Hughes

Suggested Reading

DiPALS Writing Committee on behalf of the DiPALS Study Group Collaborators. Safety and efficacy of diaphragm pacing in patients with respiratory insufficiency due to amyotrophic lateral sclerosis (DiPALS): a multicentre, open-label, randomised controlled trial. Lancet Neurol. 2015;14(9);883-892.

Glass JD, Hertzberg VS, Boulis NM, et al. Transplantation of spinal cord-derived neural stem cells for ALS: analysis of phase 1 and 2 trials. Neurology. 2016;87(4):392-400.

Petrou P, Gothelf Y, Argov Z, et al. Safety and clinical effects of mesenchymal stem cells secreting neurotrophic factor transplantation in patients with amyotrophic lateral sclerosis: results of phase 1/2 and 2a clinical trials. JAMA Neurol. 2016;73(3):337-344.

U.S. Food and Drug Administration. FDA grants accelerated approval to first drug for Duchenne muscular dystrophy. FDA News Release. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm. Accessed November 4, 2016.

Wolfe GI, Kaminski HJ, Aban IB, et al. Randomized trial of thymectomy in myasthenia gravis. N Engl J Med. 2016;375(6):511-522.

Pages

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