Three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A.
Blog Archive
Blog
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
a mutation in the SORD gene that may may affect 60,000 patients worldwide
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
Work from home NTI @Home
HNF is thrilled to present a new webinar with NTI@Home, a non-profit dedicated to providing people with disabilities and their caregivers with free virtual job training and placement
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”).
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