Healio

Partnership to advance therapeutic for Charcot-Marie-Tooth disease type 1a

Please provide your email address to receive an email when new articles are posted on . A nonprofit focused on treatments and cures for Charcot-Marie-Tooth disease will partner with a biotechnology ...
Healio

FDA grants orphan drug designation to RNA therapy for Charcot-Marie-Tooth disease type 1A

Please provide your email address to receive an email when new articles are posted on . The FDA has granted orphan drug designation to DTx-1252, an investigational small interfering RNA therapeutic to ...
EurekAlert!

Promising new treatment for a common hereditary nerve disease

Researchers from Tokyo Medical and Dental University (TMDU) develop a genome-editing technique that reduces disease-causing proteins and related issues in cells from a patient with Charcot–Marie–Tooth ...
Science Daily

Potential therapy for incurable Charcot-Marie-Tooth disease found

A potential new treatment approach for hereditary neurological disorder, the incurable Charcot-Marie-Tooth disease, has been found by researchers. Patients with Charcot-Marie-Tooth disease type 1A ...
아시아경제

ENCell Completes IND Application for EN001 Phase 2a Trial in Charcot-Marie-Tooth Disease Type 1A

ENCell announced on August 18 that it has completed a request to the Ministry of Food and Drug Safety to amend its clinical trial protocol for a Phase 1b/2a study targeting patients with Charcot-Marie ...
Nasdaq

Pharnext Announces First Patient Enrolled in the PREMIER Trial, its Pivotal Phase III Clinical Development Program of PXT3003 in Charcot-Marie-Tooth Disease Type 1A ('CMT1A')

PARIS, FRANCE / ACCESSWIRE / March 31, 2021 / Pharnext SA (FR0011191287 - ALPHA) (the 'Company'), an advanced late-stage clinical biopharmaceutical company pioneering new approaches to developing ...
Business Wire

Pharnext to Announce Top-Line Results from the Pivotal Phase 3 Trial of PXT3003 for the Treatment of Charcot-Marie-Tooth Type 1A Disease by October 2018

PARIS--(BUSINESS WIRE)--Regulatory News: Pharnext SA (Paris:ALPHA) (FR0011191287 - ALPHA), a biopharmaceutical company pioneering a new approach to the development of innovative drug combinations ...
Business Wire

PHARNEXT Collaborates with OrphanDev in Europe for Its Phase 3 Clinical Trial PLEO-CMT in CHarcot-Marie Tooth Type 1A Disease (CMT 1A)

PARIS--(BUSINESS WIRE)--Pharnext SAS today announced its collaboration with OrphanDev, a dedicated platform offering support in regulatory, methodology and logistics for rare diseases. OrphanDev’s ...
FOX31 Denver

Pharnext: Findings From 'Real-World' Digital Lifestyle Study, CMT&Me, on Symptom Burden of Charcot-Marie-Tooth Disease Type 1A Published in the Journal of Clinical ...

PARIS, FRANCE / ACCESSWIRE / September 14, 2022 / Pharnext SA (FR0011191287:ALPHA) (the "Company"), an advanced late-clinical stage biopharmaceutical company developing novel therapeutics for ...