Exploratory Phase 2a study showed consistent and clinically meaningful functional improvements across muscle strength, motor performance, and ...
A phase 2a trial of NMD Pharma’s rare disease prospect ignaseclant has missed its primary endpoint. | A phase 2a trial of NMD ...
ATLANTA, GA, USA, February 20, 2024 /EINPresswire.com/ -- The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot ...
Improvements in outcomes and reduction in sorbitol maintained through 24 months Statistically significant correlation between absolute reduction in sorbitol and change in 10MWRT and CMT-FOM Lower Limb ...
This study marks an important step forward in HNF’s mission to drive advancements in digital health technologies for CMT. With the initial success and validation of wearable devices, HNF is paving the ...
This is the 2 nd orphan drug designation granted to the skeletal muscle-specific drug therapy NMD670 Under this newly granted orphan drug designation, NMD670 is currently being evaluated in a Phase 2 ...
Researchers from Cologne, Bochum, Padova and Angers have discovered a novel connection between mitochondrial function, protein quality control and cellular health, whose failure could be the leading ...
Applied Therapeutics, Inc. announced promising 18 and 24-month results from the INSPIRE Phase 2/3 trial of govorestat, a treatment for Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD).
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