Passage Bio has acquired the rights for a gene therapy developed at the University of Pennsylvania (Penn) that aims to restore the function of the MFN2 gene, which is ... Read more
September is Charcot-Marie-Tooth (CMT) awareness month, and the Hereditary Neuropathy Foundation (HNF) suggests it is an ideal time to increase awareness — among the general public, educators, legislators, and physicians ... Read more
Pharnext is planning a new Phase 3 clinical study to further explore the potential of PXT3003 in patients with Charcot-Marie-Tooth disease type 1A (CMT1A). The decision to launch this ... Read more
Schwann cells — those responsible for the production of the myelin protective layer in peripheral nerve cells — fail to develop normally in the absence of a protein called ... Read more
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more
A new technique allows for detailed understanding of how proteins are transported in the nerve cells that connect the eye to the brain, and this may have implications for ... Read more
A mutation in the periaxin (PRX) gene has been reported for the first time as the cause of Charcot-Marie-Tooth (CMT) type 4F in a Chinese patient. The report, “Novel mutation in the ... Read more
Even if not experiencing overt clinical signs, Charcot-Marie-Tooth Disease Type 1A (CMT1A) patients able to walk show respiratory muscle weakness associated with phrenic nerve damage, according to a new ... Read more
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more
Pin It on Pinterest
We use cookies to ensure that we give you the best experience on our website. If you continue to use this site we will assume that you are happy with it. We never use your cookies for creepy ad retargeting that follows you around the web. OkRead more
