Meet Addie “The Face of the CMT Medical Journal”
New Study Measures Progression of CMT1A Nerve Impairment
New Study Measures Progression of CMT1A Nerve Impairment
New Study Measures Progression of CMT1A Nerve Impairment
Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)
Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’) New results suggest good safety profile and sustained efficacy of PXT3003 as measured with the...SORD: A Lesson in Repurposing Drugs to Treat Hereditary Neuropathy
SORD: A Lesson in Repurposing Drugs to Treat Hereditary Neuropathy GUEST AUTHOR: DAN KNAUSS SORD is a gene that has received a lot of attention lately. International research on SORD has increased greatly since the discovery in 2020 that its mutation causes the most...
TRPV4 Hereditary Neuropathy Patients Connect with Johns Hopkins Experts to Push Research Efforts Forward
BY STEPHANIE CARMODY, TRPV4 RELATED HEREDITARY NEUROPATHY (CHARCOT MARIE TOOTH TYPE 2C) PATIENT TRPV4 Hereditary Neuropathy Patients Connect with Johns Hopkins Experts to Push Research Efforts Forward When you are diagnosed with an ultrarare type of hereditary...
Mouse Models of Human CNTNAP1 Mutations: Strategies to Rescue and Restore Functions
MANZOOR A. BHAT, M.S., PH.D., DEPARTMENT OF CELLULAR AND INTEGRATIVE PHYSIOLOGY UT HEALTH SAN ANTONIO, TX Myelination of nerves has the sole purpose of allowing axons to propagate nerve impulses over long distances in a saltatory manner. Peripheral or central...
Novel findings of a new common type of CMT2 that might be the 1st step to a treatment: SORD gene deficiency, the most common autosomal-recessive type of CMT
Dr. Stephan Züchner and colleagues at the University of Miami (Drs. Andrea Cortese, Grace Zhai, Andriana Rebelo), along with the Inherited Neuropathies Consortium (INC) – a group of CMT academic experts led by Dr. Michael Shy at the University of Iowa – has...
The Global Registry for Inherited Neuropathies (GRIN) powers the Hereditary Neuropathy Foundation’s patient-centered research and drug discovery initiatives. Leading Charcot-Marie-Tooth advocacy organization’s innovative platform plays a critical role in its mission.
GRIN has played a critical role in identifying important issues related to the CMT patient experience, revealing new areas to explore and research.
$61,000 Raised to Support Pediatric CMT Trials
Last summer HNF teamed up with the Penn Medicine Orphan Disease Center (ODC) for the Million Dollar Bike Ride (MDBR) in Philadelphia. Our fundraising goal of over $30k was achieved and matched by the ODC, granting HNF over $60k to allocate to CMT research. Thanks to...
Pharnext Announces PXT3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A has Been Granted Promising Innovative Medicine (PIM) Designation by UK Medicines and Healthcare Products Regulatory Agency
PARIS, France, 8:30 a.m., March 18, 2020 (CET) – Pharnext SA (FR0011191287 – ALPHA), a biopharmaceutical company pioneering a new approach to developing innovative drug combinations based on big genomics data and artificial intelligence, today announced that the... Pharnext raises € 7.7 million in a private placement
PARIS, France, 08:30 a.m., March 05, 2020 (CET) – Pharnext SA (FR0011191287 – ALPHA) (the “Company”), a biopharmaceutical company pioneering a new approach to developing innovative drug combinations based on its PLEOTHERAPY artificial intelligence platform...
HNF on the Forefront of Pain Research to Support CMT Patients
Allison Moore, HNF Founder/CEO, along with her team, took action and led the HNF groundbreaking CMT pain initiative to help the community.