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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 Announces Strategic Partnership with Acceleron to Advance CMT Treatment Options
…treatments, and ultimately, a cure for CMT. Strategic alliances with corporate partners like Acceleron support innovative drug development with a goal to rapidly deliver therapies to CMT patients. “We are…
MDA and CMTA Partner to Advance Treatments and Care for Charcot-Marie-Tooth Disease
…is one of the neuromuscular diseases MDA fights as an umbrella organization with a big picture perspective on finding treatments and cures for kids and adults whose weakening physical strength…
Toxic Medications List For Patients with Charcot-Marie-Tooth Disease
…ill and is offered any drug treatment by their physician, concern may arise about possible drug-related side effects, and these side effects may target the peripheral nerves. It is reasonable…
Frequently Asked Questions: Ionis/CMTA 1A Research Announcement
…measures, there was an overall improvement that was observed in both animal models with ASO treatment. Another important part of this study was to evaluate ASO treated mice and rats…
Medication Study
…lithium, and pyridoxine. Overall, Weimer and Podwall conclude that treatment with vincristine poses an “unacceptable risk to patients with known or possible CMT1A.” They further conclude that the use of…
Research in Charcot–Marie–Tooth Disease Type 1A (CMT1A)
…over $200,000 invested, we’re pioneering methods to evaluate potential treatments targeting myelin defects in CMT1A. Join us as we revolutionize CMT understanding and treatment, contributing to a larger framework of…
Type 1 CMT Gene Therapy Project Now Underway
…CMT (type 1 and most type 4).” “Gene therapy continues to show promise for the treatment of neuromuscular disease,” said Amanda Haidet-Phillips, Ph.D., one of the MDA’s scientific portfolio directors….
Taysha Gene Therapies & CMTA Collaborate to Increase Genetic Testing Access for Giant Axonal Neuropathy (GAN)
…TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare…
CMT2A Research
…reagents can prevent disease pathology in a rodent model of CMT2A. PROJECT GOAL: EARLY PHASE TREATMENT TRIALS OF CMT2A Grant Amount: $18,942 Principal Investigator: Michael Shy, MD, University of Iowa…