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…
Webinar Recordings
…spent the last decade specializing in treatment of adults with acquired neurological impairments. She is certified in neuro-developmental treatment and the use of physical agent modalities. She has specialty training…
Applied Therapeutics Announces Positive Sorbitol Reduction Data From the Ongoing Phase 3 INSPIRE Trial in Sorbitol Dehydrogenase (SORD) Deficiency
…a pre-specified interim analysis of the ongoing Phase 3 INSPIRE trial, AT-007 reduced sorbitol levels by a mean of approximately 52% (or approximately 16,000ng/ml) over 90 days of treatment (p…
CMTA’s 40 Under 40
…a pivotal role in driving progress towards effective treatments and, ultimately, a more promising future for the CMT community. Trained by their elders, these young individuals are pushing the boundaries…
Initiation of Registrational Phase 2/3 Study of AT-007 in SORD Deficiency
…Deficiency patients, AT-007 reduced blood sorbitol levels by approximately 66% from baseline through 30 days of treatment. The range of reduction from baseline in patients was 54%-75%. AT-007 was safe…
CMTA and Pharnext Enter Biomarker Research Collaboration
…is currently no treatment available.” About Pharnext Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapeutics for orphan and common neurodegenerative diseases that currently lack curative and/or disease-modifying treatments….
CMTA-STAR Biotech & Alliance Partners
…the development of innovative therapeutics harnessing the Integrated Stress Response (ISR) for the treatment of a broad range of diseases. The company plans to demonstrate the clinical effectiveness of its…