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, 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…
…the treatment with their physician to see if other precautions or preventative treatments are needed. I am treating a 20-year-old patient with leukemia. After his fourth dose of vincristine, he…
…bound with limited leg movement may be at higher risk and should discuss the treatment with their physician to see if other precautions or preventative treatments are needed. I am…
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 GLENOLDEN, PENNSYLVANIA, USA, NOVEMBER 3, 2022 — Shift Pharmaceuticals, a privately held company that is developing antisense oligonucleotides (ASOs) to treat…
…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…
…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…
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…
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…