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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…
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…
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…
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…
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…
Shift Pharmaceuticals, CMTA Partner on ASO Treatment for CMT1A
Shift Pharmaceuticals, CMTA Partner on ASO Treatment for CMT1A GLENOLDEN, PENNSYLVANIA, USA, NOVEMBER 3, 2022 — Shift Pharmaceuticals, a privately held company that is developing antisense oligonucleotides (ASOs) to treat…
Consensus_Statement_on_Surgical_Treatment_of_CMT
…the success or fail- ure of their treatment? A history of genetic testing should be noted. Although not required for surgical treatment, the CMT genotype may be helpful in better…
CMT1A Ionis Press Release
New Research Shows Promise for CMT Progressive Neuromuscular Disease Potential treatment offers hope for over 2.8 million affected worldwide Glenolden PA, December 4, 2017—A new paper published December 4th on…
Data Shows Benefit with PXT3003 in Patients with CMT1A
…treatments. Pharnext has two lead products in clinical development. PXT3003 completed an international Phase III trial with encouraging topline results for the treatment of Charcot-Marie-Tooth disease type 1A (‘CMT1A’) and…
A Cure for Caroline and Other CMT Stories
Caroline’s journey with a rare and serious form of CMT began when she was just 9 months old. Today, a cure for Caroline may open the door to treatments and…