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.
Maybe it will work on CMT..?
Category: News
News about Charcot Marie Tooth (CMT), neuropathic disorders, and neuromuscular diseases.
New discovery of a genetic cause (and likely cure) for a previously unknown subtype of CMT2
Published in Nature Genetics last month, the INC group's findings are a big deal, and there is a really cool story from the University of Miami's Miller School of Medicine about how the research team came together.
CMT, COVID-19, and Respiratory Health
A few weekends ago, I listened to the HNF webinar on COVID-19 and CMT with Dr. John Bach of Rutgers University Medical School, a leader in the field of pulmonary rehabilitation, particularly for people with neuromuscular diseases. You can watch it and get a bunch of other materials now — for free — thanks to the HNF.
ACE-083 Trials Show no Benefit
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.
Video short on CMT – Rare Film Festival
This is a great, short video that explains the basics of CMT — made by students at the University of Greenwich for the first "Rare Film Festival," put on by Rare Disease UK.
New genetic diagnostics for CMT
Here's an informative article from the Hereditary Neuropathy Foundation (HNF) about current best practices in genetic diagnosis of CMT. A lot of examples are given of people with less common subtypes who waited a long time to get properly diagnosed — sometimes after many years of wrong diagnoses or none at all. Today the best… Continue reading New genetic diagnostics for CMT
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.
Play the didgeridoo (or harmonica) to fight sleep apnea
Some recent studies on obstructive sleep apnea got attention for concluding something fairly obvious — losing weight and reducing fat in the tongue might be the best treatment for OSA.
