a mutation in the SORD gene that may may affect 60,000 patients worldwide
Author Archive
Estela Lugo
Painting By Mouth – Kaileen Selig
CMT doesn’t stop artist, Kaileen Selig from painting beautiful pieces using her mouth.
GRIN powers HNF’s patient-centered research and drug discovery initiatives.
GRIN has played a critical role in identifying important issues related to the CMT patient experience, revealing new areas to explore and research.
Meet Dr. Nivedita Jerath, MD, MS – AdventHealth Neuroscience Institute Center of Excellence
The AdventHealth Neuroscience Institute is fully committed to patient excellence, care, and exceeding expectations. Patients will experience a team that is superb beyond expectations. If they are coming from far, they will be catered to with our destination team. When they arrive, they will experience an environment full of love, kindness, friendliness, and softness. They will observe an amazing facility, receive an in-depth physician evaluation, electrodiagnostic testing, genetic testing, and opportunities to get all the multidisciplinary therapies that they need. They will also be involved in any research studies that we may have.
Spinning 4 CMT: My CMT Story
Marc Daigle’s CMT story
Rishi Sharma CMT 2A Wheelchair Rugby
Rishi’s CMT story
Meet the Kohler Family: Alana has GDAP1
Alana Kohler’s story with CMT4A.
$61,000 Raised to Support Pediatric CMT Trials
Last summer HNF teamed up with the Penn Medicine Orphan Disease Center for the Million Dollar Bike Ride in Philadelphia.
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
United Kingdom’s Medicine and Healthcare products Regulatory Agency (MHRA) has granted Promising Innovative Medicine (PIM) designation to its lead drug candidate, PXT3003, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A) in patients 16 years and older.
Pharnext raises € 7.7 million in a private placement
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 harnessing big genomics data and network pharmacology, today announced a capital raise of circa € 7.7 million by way of issuance of 1,799,061 new ordinary shares (the “New Shares”) with one warrant attached each (together with the New Shares, the “ABSA”).
ACE-083 did not achieve statistically significant improvements in functional endpoints relative to placebo; Acceleron to discontinue development of ACE-083.
treatment with ACE-083 in patients with Charcot-Marie-Tooth disease (CMT) did not demonstrate functional improvement in the Phase 2 trial
The Hereditary Neuropathy Foundation’s Movement is Medicine™ Program Expands to Florida and Continues to Shed Light on the Impact Exercise Has on Charcot-Marie-Tooth (CMT)
The Hereditary Neuropathy Foundation’s Movement is Medicine™ Program Expands to Florida and Continues to Shed Light on the Impact Exercise Has on Charcot-Marie-Tooth (CMT)
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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)
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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)
CMT Type – CMT1B (2)
CMT Type – CMT1X (3)
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CMT Type – HDAC6 (1)
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CMT Type – SORD Deficiency (9)
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