CATEGORIES: CMT Update | GRIN Patient Registry | Research and Critical Trials | TRIAD

Hot off the Press: CMT Update

by Courtney | Feb 23, 2023 | 0 comments

As we start the new year energized and ready to continue our mission of bringing treatments and cures for CMT, we have rebranded our CMT Update newsletter to feature our lead research programs including the GRIN patient registry, the HNF-funded research milestones that have been met, and how patients can continue to participate in the process. The CMT Update will continue to be published quarterly.

Winter 2023 CMT Update

HNF is Reshaping Drug Repurposing for CMT
Pharnext Unveils the Latest Progress
Is this Symptom Related to CMT?
Join the Global Registry for Inherited Neuropathies
CMT Genie and more…

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Good News for CMT1A Patients – PXT3003

by Estela Lugo | Jun 9, 2020 | CMT Type - CMT1A, Research, TRIAD | 0 Comments

Three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A.

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

by Estela Lugo | Jun 7, 2020 | Applied Therapeutics, CMT Type - SORD Deficiency, Research | 7 Comments

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.

by Estela Lugo | Apr 15, 2020 | Featured, GRIN Patient Registry, Research, TRIAD | 2 Comments

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

by Estela Lugo | Apr 9, 2020 | Pediatrics & CMT, Research, TRIAD | 1 Comment

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

by Estela Lugo | Mar 18, 2020 | Pharnext, Research | 5 Comments

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

by Estela Lugo | Mar 11, 2020 | CMT Type - CMT1A, Research, TRIAD | 0 Comments

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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Hot off the Press: CMT Update

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