Tag: CMT1A

• Maybe it will work on CMT..?

  

  It seems the new hope for VM202 as a CMT1a treatment is perhaps Hellxmith’s latest attempt to find a market for an experimental product that has not panned out as hoped.

  Dan

  July 27, 2020

  News, Research

  ALS, arterial disease, clinical trials, CMT1A, diabetes, gene therapy, genetics, Helixmith, HGF, Lyrica, paracrine signalling, plasmid, VM202

• CMT and Respiratory Dysfunction

  

  Science is starting to confirm very wide ranging effects to the nerve damage CMT does, often slowly and over time so it may not be very noticeable for many years.

  Dan

  March 11, 2020

  Research, Therapy

  Breathing, CMT and Respiratory Impairment, CMT1A, CMT2, CMTNS, Hypercapnia, Hypercarbia, Lungs, MEP, MIP, Respiration

• Second Phase 3 Clinical Trial for CMT1A “Pleodrug”

  

  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.

  Dan

  February 17, 2020

  News, Research

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

• Sleep Apnea and CMT

  

  Studies show CMT tends to come with Disturbed Sleep, Depression, and Reduced Quality of Life. 😞 This post summarizes the scientific research I’ve absorbed on the subject of CMT and sleep apnea. It’s definitely accurate relative to my own experience in the past year or so.

  Dan

  January 4, 2020

  Research, Therapy

  Asthma, BiPAP, Blood Oxygen, CMT1, CMT1A, CMT2, CMT2C, CPAP, Depression, Hypercapnia, Hypercarbia, Hypoxemia, Larynx, Pharynx, Quality of Life, Sleep Apnea, Vocal Cords

• The State of Research for Cures (2019)

  

  The US-based CMTA (Charcot Marie Tooth Association) has a 12-year-old program called the Strategy to Accelerate Research (STAR) that is actively pursuing the causes and possible cures for CMT. STAR will cover nearly all CMT Type 1s and 2s, including types with as yet unidentified genetic causes.

  Dan

  December 31, 2019

  Research

  Antisense Oligonucleotides, Axon, CMT1, CMT1A, CMT1B, CMT1X, CMT2, CMT2A, CMT2E, CMT4, CMT4C, CMTX, CRISPR, Inherited Neuropathies Consortium, Ionis Pharmaceuticals, Kleopas Kleopa, Mitofusin 2, Muscular Dystrophy, Myelin, Neuromuscular Disorders, PMP22, Schwann Cells, Spinal Muscular Atrophy, STAR, Stephan Züchner