Helixmith, specializing in gene therapy research for over 20 years, has kicked off its phase I and 2a clinical trial for using VM202 (Engensis) to treat CMT1A.
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Clinical Trials
Total posts in Category: 21
Press Release: Pharnext provides regulatory and clinical update on PXT3003 Phase III study for the treatment of Charcot-Marie-Tooth Type 1A
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
ACE-083 did not achieve statistically significant improvements in functional endpoints relative to placebo; Acceleron to discontinue development of ACE-083.
treatment with ACE-083 in patients with Charcot-Marie-Tooth disease (CMT) did not demonstrate functional improvement in the Phase 2 trial
HNF’s Roadmap To Clinical Trial Development
2018 will be “the year” for our community as we prepare for the externally-led Patient-Focused Drug Development Meeting.
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.
Are You a Patient With CMT 1A? New Clinical Trial Opportunity Available For You!
For the first time investigators are looking for patients to participate in a pivotal Phase 3 clinical trial of Pharnext’s lead investigational pleodrug, PXT-3003 for the potential treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Pharnext Opens First U.S. Study Site For Its PLEO-CMT International Pivotal Phase 3 Trial In Charcot-Marie-Tooth Disease Type 1A
French pharmaceutical company Pharnext announced the opening of the first U.S. trial site for its PLEO-CMT pivotal Phase 3 clinical trial of its lead pleodrug PXT-3003 in Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Breaking News: First Therapeutic Gene Therapy to Treat an Inherited Neuropathy is Approved for Clinical Trial!
The first disease community to receive a therapeutic gene to the spinal cord for an ultra rare inherited neuropathy is Giant Axonal Neuropathy (GAN). Congratulations to Hannah’s Hope Fund (HHF), a 501(c)3 public charity, which has driven this collaborative research in less than six years. Six million dollars has been raised to date to fund pre-clinical and clinical research on this rare disease.
Positioning HNF to Translate Treatments to Clinical Trials
In June 2013, HNF made the important decision to refine the organization’s mission. We now place more importance on new initiatives that take our research discoveries and translate them to the next phase in the drug discovery process.
We divide our research initiatives into 3 areas:
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