The Hereditary Neuropathy Foundation and Acceleron Pharma have been collaborating since January, 2016. Acceleron Pharma is a ”clinical stage biopharmaceutical company“ based in Boston, Massachusetts.

Their lead investigational drug is based on a naturally occurring protein and works to enhance the body’s own promoters of muscle growth. ACE-083 can be customized to patients needs by administering the drug into target muscle(s) with the aim of increasing muscle size and strength. The drug was developed for neuromuscular diseases in which patients experience weakness and atrophy in specific muscles, such as CMT patients with foot drop.

  • August, 2016 conducted focus groups in Cambridge, MA at Acceleron Pharma headquarters to help with the clinical trial design of their ACE-083 phase II study
  • February, 2017 launched a collaborative Patient Reported Outcomes (PRO) study* to help Acceleron Pharma gain additional insight into the burden of the disease from the patient perspective
  • July, 2017 Allison Moore presented the “CMT Patient Experience and the Impact of Patient Reported Outcomes Study on Clinical Research and Trials” at the Peripheral Nerve Society Meeting in Barcelona, Spain  
  • September, 2018 HNF sheds light on the impact foot drop has on CMT patients’ quality of life at the Externally-led PFDD Meeting to inform the FDA on the importance of a drug that may mitigate this debilitating symptom

“People suffering from CMT currently have no drug therapy options to address the major consequences of this serious disease, such as foot drop leading to mobility impairment and frequent falls. We hope that ACE-083 will prove to be an important agent to increase lower leg function, improve the ability to walk, and reduce falls.” — Robert K. Zeldin, M.D., Chief Medical Officer of Acceleron.

For more information on the randomized, double-blind placebo controlled study using the optimal dose level selected from Phase II part 1 for CMT1 and CMTX of this study, can be found on, identifier NCT03124459.

*This study is being referenced globally by CMT researchers, clinicians, and industry at conferences accepted as poster abstracts, oral presentations and was highlighted at the groundbreaking Externally-led PFDD Meeting.

acceleron meeting