Pediatric CMT Natural History Study enhancement to their Charcot-Marie-Tooth (CMT) and Inherited Neuropathies (IN) patient registry, Global Registry for Inherited Neuropathies (GRIN).
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Research
Total posts in Category: 95
First Ever Biorepository For Charcot-Marie-Tooth, HNF Launches The CMT Biobank
The Hereditary Neuropathy Foundation (HNF), an advocacy and research 501c3 non-profit, today...
DTx Pharma Receives FDA Orphan Drug Designation for DTx-1252 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Dtx today announced that the FDA has granted Orphan Drug Designation to DTx-1252, an investigational FALCON small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Great News From HNF TRIAD Industry Partner — Pharnext
Pharnext announces new positive results from the ongoing open-label extension study of their drug PXT3003
Great News From HNF TRIAD Industry Partner — Applied Therapeutics
Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation
HNF sheds Light & Research Funding on Mitochondrial Diseases Linked to CMT
Mitochondria are the powerhouses of our cells. Think of them as our body’s batteries. Mitochondrial disease causes these batteries to run low.
Hereditary Neuropathy Foundation Re-Launches One-of-A-Kind Patient Registry for Charcot-Marie-Tooth Disease Research
Hereditary Neuropathy Foundation Re-Launches One-of-A-Kind Patient Registry for...
Battersby Lab Donor Story: Fighter Moms Donate to Vital Research into Mitochondrial Diseases
Battersby Lab Donor Story Fighter Moms Donate to Vital Research into Mitochondrial Diseases
SORD INSPIRE Phase III Clinical Trial
HNF Partner Applied Therapeutics is Currently Enrolling SORD Deficiency Patients for the INSPIRE Phase III Clinical Trial
3 New CMT Drug Development Projects
HNF Announces 3 New Drug Partnerships & Pathways Targeting Multiple Forms of CMT.
What is SORD Deficiency: Part 2
Join us for this webinar as we learn more about Sorbitol Dehydrogenase (SORD) Deficiency
HNF collaborates with Rarebase on a Drug Discovery Platform to develop treatments for Charcot-Marie-Tooth (CMT)
HNF collaborates with Rarebase on a Drug Discovery Platform to develop treatments for Charcot-Marie-Tooth (CMT)
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