The National Organization for Rare Diseases has awarded HNF funding to build a robust natural history study to support the second most common type of CMT called hereditary neuropathy pressure palsies (HNPP).
Q&A with Dave Washabaugh
HNF has launched a new Patient-Reported Research Study to advance therapies for CMT and inherited neuropathies (IN) in an effort to collect comprehensive data for a better understanding of the disease.
HNF’s initial gene therapy work will focus on CMT type 6, which is caused by a recessive mutation in the C12orf65 gene and is currently no treatment.
video of Robert’s keynote about CMT going largely unrecognized