Hear from Allison Moore, Founder/CEO of the Hereditary Neuropathy Foundation about the past, present and future or CMT research.
A new type of Charcot-Marie-Tooth (CMT) has recently been identified caused by mutations in the SORD gene.
What GRIN is and why you should join
Participating during CMT month
The FDA has agreed with the key elements of Pharnext’s approach for the developmental pathway to approval for PXT3003.
Alana Kohler’s story of having CMT4A.
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
Three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A.
a mutation in the SORD gene that may may affect 60,000 patients worldwide
GRIN has played a critical role in identifying important issues related to the CMT patient experience, revealing new areas to explore and research.
Last summer HNF teamed up with the Penn Medicine Orphan Disease Center for the Million Dollar Bike Ride in Philadelphia.
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