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MDA and CMTA Fund Grant to Study Gene Therapy in Charcot-Marie-Tooth Disease
…whether treatment at later stages of the disease leads to improvements similar to those seen for treatment in the early stages. CMTA CEO Gilles Bouchard said, “Partnerships are at the…
Coronavirus (COVID-19) and CMT
…impacts will the vaccine have on possible future gene therapy treatments or medications? Whether the vaccine will have an adverse impact on any course of treatment that you or your…
Voice_of_the_Patient_Report_8-19-2019
…meeting, a parallel effort to FDA’s PFDD initiative to more systematically obtain patients’ perspectives on the burden of disease and impact of current treatments or lack thereof in Hyattsville, Maryland…
CMTA Awards $225,483 for Study On Using ASOs to Treat CMT2E
…a functional protein. They are short man-made pieces of genetic material designed to bind to a specific target messenger, RNA (mRNA). The Food and Drug Administration has approved ASO-based treatments…
CMTA Hails Novartis Acquisition of DTx Pharma And Its CMT1A Therapeutic Program
…of novel CMT treatments capable of reaching the peripheral nervous system. Under the terms of the agreement, Novartis will make an upfront payment of $500 million and additional payments of…
Understanding Charcot-Marie-Tooth Disease in Children
…no approved treatments for CMT. Understanding CMT in Children Children with CMT sometimes take longer to reach physical milestones, such as walking, hopping, jumping with two feet, or getting up…
CMTA Alliance Partner, Applied Therapeutics, Announces Positive 12-Month Results in its Ongoing INSPIRE Phase III Trial for CMT-SORD
…statement, Applied Therapeutics said the trial at its 12-month mark has shown a statistically significant correlation between sorbitol levels and the prespecified CMT Functional Outcomes Measure (CMT-FOM) and that treatment…
October 2023 Community Statement from Novartis
“The CMTA welcomes this community update from Novartis and applauds its leading role in the development of treatments for CMT. The CMTA has been maintaining regular contact, providing support and…
CMTA Appoints Biotech Industry Veteran Suzanne Bruhn, PhD as New CEO
…treatments for rare diseases, will take the helm of the patient-led nonprofit as it moves to the next phase of its search for a cure for CMT. Dr. Bruhn brings…
Pharnext Announces Disappointing Results for CMT1A Drug Trial PXT-3003
…can be influenced by placebo and training effects, making it difficult to determine the impact of treatment. In particular, the company said, the Overall Neuropathy Limitation Scale (ONLS), which measures…