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Living and Learning with Charcot Marie Tooth Disease.

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Tag: ACE-083

ACE-083 was a drug developed by Acceleron Pharma Inc. to increase muscle mass in people with muscle atrophy from MD and CMT. In 2019-20, clinical trials showed insufficient benefits to continue development of the drug.

ACE-083 Trials Show no Benefit

March 12, 2020March 13, 2020 DanLeave a comment

Acceleron Pharma Inc's ACE-083 drug trials "did not achieve statistically significant improvements in functional endpoints relative to placebo" with CMT patients, so it will no longer be developed.

Posted in News, ResearchTagged Acceleron, ACE-083

Second Phase 3 Clinical Trial for CMT1A “Pleodrug”

February 17, 2020March 23, 2020 DanLeave a comment

After the setback of having to do a second Phase 3 clinical trial, Pharnext's ongoing drug trials for PXT3003 show positive results in people with CMT1A.

Posted in News, ResearchTagged Acceleron, ACE-083, Baclofen, clinical trials, CMT1A, D-sorbitol, Daniel Cohen, naltrexone hydrochloride, ONLS, Pharnext, pleodrug, pleotherapy, PMP22, PXT3003, Rodolphe Hajj

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Acceleron ACE-083 Axon BiPAP Breathing clinical trials CMT1 CMT1A CMT2 CMTA CMT and Respiratory Impairment Covid-19 CPAP D-sorbitol Depression diabetes Disability genetics HNF Hypercapnia Hypercarbia Lungs Mental Health MFN2 PMP22 Quality of Life Respiration Respiratory Health Sleep Apnea Stephan Züchner

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