Three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A.
Blog Category
Research
Total posts in Category: 95
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
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.
$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”).
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.
Potential Novel Treatment for a Rare Form of Charcot-Marie-Tooth (CMT) Neuropathy
In 1998 Dr. Florian Thomas met a five-generation family with hereditary neuropathy leading to a research effort now entering its third decade.
CMT&ME APP Adds New Medical Profile
The CMT&ME app has updated the research app to include a brand new medical profile.
HNF tackles the hidden mysteries behind the genetic diagnosis of CMT mitochondrial diseases
C12orf65 gene, a rare form of CMT also known as Leigh Syndrome is robbing young children of a normal life even beyond the classic CMT symptoms.
Won’t You Please Consider A Year End Gift?
Learn the top 15 ways the Hereditary Neuropathy Foundation put your generous donations to work for you this year.
PNS, It’s A Global Effort
Where do the top CMT researchers in the world unite to share leading-edge research? The Peripheral Nerve Society (PNS) meeting!
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Faces of CMT – Grace (18)
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)
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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)
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Burke Insitute (3)
University of Cambridge (1)
University of Miami (1)
University of San Antonio Texas (1)
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Applied Therapeutics (10)
Pharnext (10)
Rarebase (4)
Types of CMT (1)
CMT Type – CMT1A (22)
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