EN001, a novel therapy under development by ENCell, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Charcot-Marie-Tooth (CMT) disease. Phase 1 clinical trial confirmed ...

ENCell’s investigational drug EN001 has been granted Orphan Drug Designation by the FDA for the treatment of ...

Charcot-Marie-Tooth (CMT) disease, a group of heritable disorders that affect the peripheral nerves, is marked by specific genetic changes. Research by the team of Prof. Ludo Van Den Bosch (VIB-KU ...

This discovery opens new possibilities for treating diseases like CMT. "Although MFN2 is a leading causative gene in Charcot-Marie-Tooth, most other genes linked to the disease do not encode ...

This discovery opens new possibilities for treating diseases like CMT. Although MFN2 is a leading causative gene in Charcot-Marie-Tooth, most other genes linked to the disease do not encode ...

A new study involving UCL researchers has found 69 previously unidentified genetic determinants of rare disease, including ...

Researchers from Cologne, Bochum, Padova and Angers have discovered a novel connection between mitochondrial function, protein quality control and cellular health, whose failure could be the leading ...

Become one of the first Fully Certified CMT-FOM Clinical Evaluators at the upcoming HNF Clinical Trial Readiness Summit in Nashvil ...

This discovery opens new possibilities for treating diseases like CMT. "Although MFN2 is a leading causative gene in Charcot-Marie-Tooth, most other genes linked to the disease do not encode ...

for the treatment of TRPV4-positive Charcot Marie Tooth Disease subtype 2C (CMT2C), a severe peripheral nerve disorder. “People living with the subtype 2C of CMT, a genetically defined ...