HNF, in partnership with Rarebase, is leading the charge in the first-ever research initiative to tackle multiple types of CMT in one project using its tech-enabled drug discovery platform called “Function.”
Blog Category
Types of CMT
Total posts in Category: 1
SORD Webinar – Part 2 – Clinical Trial Update
Clinical Trial Update for SORD Deficiency – Applied Therapeutics – Dr. Shoshana Shendelman
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
Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Jaxson’s Clay Shoot 2021
Jaxson’s Clay Shoot 2021 Image gallery
“Grow or Mow” A Sheer Success!
Brandon, his wife, and Alana would host a virtual ‘Mow or Grow’ fundraiser with each $1 vote going towards CMT research.
New Study Measures Progression of CMT1A Nerve Impairment
New Study Measures Progression of CMT1A Nerve Impairment
Interim Analysis Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)
Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)
SORD: A Lesson in Repurposing Drugs to Treat Hereditary Neuropathy
Research from a mutation on the SORD gene
TRPV4 Hereditary Neuropathy Patients Connect with Johns Hopkins Experts to Push Research Efforts Forward
Community connected with the TRPV4 HN team at Johns Hopkins
CMT1A Clinical Trial Update: Pharnext PREMIER trial of PXT3003
The PREMIER Trial, which is being conducted in patients with mild-to-moderate CMT1A, is expected to enroll approximately 350 subjects ages 16-65 with a confirmed genetic diagnosis of CMT1A.
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Faces of CMT – HELP Fund (8)
Faces of CMT – HNPP Fund (3)
Faces of CMT – James (1)
Faces of CMT – Jaxson (7)
Faces of CMT – Zach (18)
Featured (28)
GRIN Patient Registry (39)
HNF Team (10)
Industry (3)
Living with CMT (78)
Medical professionals (2)
Movement Is Medicine (17)
Optic Hereditary Neuropathy (5)
Our Impact (3)
Clinical Trials (21)
PFDD (1)
PFDD Meeting 28SEP2018 (5)
Past Events (35)
Patience Resources (0)
Accessible Travel (4)
Bracing (11)
Breathing (3)
Canine Companions (5)
Cannabis (8)
Caregivers (3)
Dating and Relationships (7)
Emotional and Mental Health (9)
Family Planning (1)
Fashion and Products (1)
Genetic Testing (11)
Home Accessibility (2)
Inclusive Employment (3)
Inspire Community (7)
Legal Rights and Benefits (8)
Neurotoxic Drugs (4)
Newly Diagnosed (7)
Nutrition (3)
Occupational Thearpy (2)
Pain (8)
Podiatry (4)
PT and Exercise (25)
Research and Critical Trials (29)
School & College (6)
Surgery (10)
Telemedicine (1)
Tips and Hacks (2)
Patient-Focused Research (1)
Pediatrics & CMT (10)
Registry (16)
Research (95)
School Outreach Program and Team CMT Kids (1)
Special Events (2)
Bike New York (19)
Other Events (4)
Spin-a-thon (7)
TCS New York City Marathon (7)
Team CMT Members (61)
The CMT Genie (6)
TRIAD (28)
TRIAD – Clinical Trial Readiness (5)
Vitaccess (1)
TRIAD – Research Gifts (0)
University of Helsinki (2)
Veneto (1)
TRIAD – Sponsored Research (0)
Burke Insitute (3)
University of Cambridge (1)
University of Miami (1)
University of San Antonio Texas (1)
TRIAD – Therapeutics (4)
Applied Therapeutics (10)
Pharnext (10)
Rarebase (4)
Types of CMT (1)
CMT Type – CMT1A (22)
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CMT Type – CMT4A GDAP1 (3)
CMT Type – CNTNAP1 (1)
CMT Type – GAN (5)
CMT Type – HDAC6 (1)
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