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Orphan Drug Designation for TSHA-120 for Treatment of GAN
Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-120 for the Treatment of Giant Axon Neuropathy (GAN) Clinical efficacy data for TSHA-120 provide quantitative evidence of…
ToolGen CMT1A Treatment Receives Orphan Drug Designation
…central part of the CMTA’s strategy to accelerate treatments for the CMT community. We congratulate ToolGen on receiving Orphan Drug Designation for its CMT1A treatment candidate and will continue to…
CMTA Alliance Partner Applied Therapeutics Plans to Submit an NDA for CMT-SORD, a Potential First Approval for the Treatment of any type of CMT
…transformative treatments for rare diseases, and we continue to work closely with regulatory agencies and patient advocacy groups to ensure that treatments become available for patients with these debilitating diseases.”…
Development of Treatments for CMT2D
…response and the CMT symptoms improved. The drug also has benefits when treatment is started after CMT symptoms have developed, and maintaining the benefits requires continued treatment. If treatment is…
Regenacy Pharmaceuticals Announces Collaboration with the CMTA to Advance Ricolinostat for the Treatment of Hereditary Neuropathy
…Regenacy Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company developing breakthrough treatments for diabetic and other peripheral neuropathies, today announced a collaboration with the Charcot-Marie-Tooth Association (CMTA), a registered non-profit organization serving…
A Comprehensive Guide to Clinical Trials
…is. In a clinical trial, participants are assigned to either receive the new treatment or a placebo treatment for comparison. In the United States, trials are conducted under strict guidelines…
DNA-Based Therapeutics: A Promising Future for CMT2E Treatment
…targeted, effective treatments for CMT, significantly improving patient outcomes. This advancement highlights the promise of precision medicine in revolutionizing the management of CMT by tailoring treatments to the genetic profile…
Novartis Secures $1.3B Deal for CMT Treatment CKD-510
Novartis is making significant strides in the field of Charcot-Marie-Tooth (CMT) disease treatments, having recently entered into a substantial $1.3 billion deal with Chong Kun Dang Pharmaceutical Corp (CKD). The…
A New Treatment for CMT1A
DTx Pharma is directly targeting the genetic cause for CMT1A in its upcoming clinical development program. DTx Pharma is developing a treatment for CMT1A that directly targets the cause of…
Charcot–Marie–Tooth Disease and Breathing Problems
…CMT-related respiratory muscle weakness provides treatment challenges the general public doesn’t typically have. Successful treatment with any of these three options rests on the individual having normal respiratory muscle strength—no…