Three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A.
Blog Category
Types of CMT
Total posts in Category: 1
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
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”).
Calling all Grandparents (and Parents)!
Join Iris Adler as she continues fundraising for her grandson and the many others living with Charcot-Marie-Tooth Type 2A
Why I’ll be attending the “Voice of the Patient Weekend” A Patient’s Perspective
I am attending HNF’s “Voice of the Patient” Weekend 2018 to tell the FDA and other stakeholders how TRPV4 HN impacts my life.
Pharnext’s lead PLEODRUG © for treatment of CMT1
Pharnext completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A.
Hereditary Neuropathy Foundation Announces Patient-Centered CMT/HNPP Pain Summit
HNF is proud to announce that our annual CMT Summit will include the HNPP community Friday, November 3, 2017, in Cambridge, Massachusetts.
HNPP Study Available for HNF Members
This study, which aims to uncover the rather unrecognized symptoms of Hereditary Neuropathy with Liability to Pressure Palsy (HNPP)
January 27, 2017 – Bridge/Brunch Fundraiser
Charity Card Party Brunch and Boutique
Please join us January 27, 2017 at 10:00 at the Broken Sound Country Club, Boca Raton Florida. Join us for an exquisite brunch and card party to benefit the H.E.L.P fund/HNF at Broken Sound Country Club in Boca Raton, FL.
Summit Speaker Spotlight: Lori Sames of Hannah’s Hope Fund
HNF has the honor of knowing one such GAN patient, Hannah Sames. She was diagnosed with GAN at four years old, and now at age eleven, Hannah can no longer walk on her own.
CBS News Report: A Mother’s Quest To Find Cure For Rare Genetic Defect
Raising global awareness of inherited neuropathies is a daily challenge we face head on at The...
Save the Date: Friday January 29, 2016 Boca Raton, Florida to Benefit CMT Type 2
Charity Card Party Brunch and Boutique Please join us January 29, 2016 at 10:30 at Broken...
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