A group is advocating for more federal money for states like Florida to boost their newborn screening for rare diseases to keep more children alive.
The MESA data show stabilization of function in participants on sevasemten versus the predicted functional decline observed in Becker natural history ...
Benitec Executive Chairman and Chief Executive Officer, Jerel A. Banks, M.D., Ph.D., will share an updated presentation detailing the significant, durable, improvements in dysphagic symptom burden and ...
Xenon shares skyrocketed nearly 50% on results that handily beat Wall Street expectations. Elsewhere, Capricor got a decision ...
Apitegromab improved motor function in SMA patients in a Phase 3 trial analysis, with stronger benefits seen in those treated earlier.