CATEGORIES: GRIN Patient Registry | Research | Research and Critical Trials

CMT Biobank: We Need Your HELP!

by Courtney | Feb 1, 2024 | 0 comments

HNF is excited to continue collecting samples for researchers and industry to help develop treatments for CMT. CMT impacts the quality of life starting in childhood and is progressively debilitating. Currently, there are no treatments, but there are many potential therapies in the pipeline. There are still gaps in understanding the natural history of the disease correlation of genotype/ phenotype, the availability of patient biospecimens for translational research, and the validation of drug candidates and biomarkers for CMT.

HNF is the only CMT advocacy group that gives access to these patient samples at minimal cost.

The locations for specimen collection are currently being discussed. Please give us feedback if you would like us to come to a city near you!

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US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III study

by Estela Lugo | Sep 15, 2020 | CMT Type - CMT1A, Pharnext, Research, TRIAD | 0 Comments

The FDA has agreed with the key elements of Pharnext’s approach for the developmental pathway to approval for PXT3003.

THERAPEUTIC RESEARCH IN ACCELERATED DISCOVERY (TRIAD) PROGRAM REQUEST FOR PROPOSAL GUIDELINES

by Estela Lugo | Sep 9, 2020 | Research | 0 Comments

The Hereditary Neuropathy Foundation (HNF) would like to advance preclinical research as quickly as possible if it aligns with our key objectives.

Accelerate Clinical Trials in Charcot-Marie-Tooth Disease (ACT-CMT) Calling CMT1A Patients Ages 18-75

by Estela Lugo | Sep 1, 2020 | Clinical Trials, Research | 2 Comments

This study is a new international research project funded by the National Institutes of Health (NIH) called Accelerate Clinical Trials in Charcot-Marie-Tooth Disease (ACT-CMT).

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”).

HNF on the Forefront of Pain Research to Support CMT Patients

by Estela Lugo | Jan 9, 2020 | Research, TRIAD | 0 Comments

Allison Moore, HNF Founder/CEO, along with her team, took action and led the HNF groundbreaking CMT pain initiative to help the community.

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CMT Biobank: We Need Your HELP!

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