Summit Therapeutics plc has announced encouraging interim results from its phase 2 PhaseOut DMD clinical trial that suggest the MDA research grant-funded experimental treatment ezutromid (formerly SMT C1100) potentially may be effective as a treatment for DMD.
Researchers are looking for individuals with SMA type 2 to participate in the phase 1 STRONG clinical trial, to test safety, dosing and proof of concept for efficacy for its SMA gene replacement therapy AVXS-101.
Researchers are looking for people with MG to participate in a phase 2 clinical trial, to test the experimental drug RA101495 in people with generalized MG who are positive for acetylcholine receptor autoantibodies.
Solid Biosciences announced the launch of its first clinical trial for SGT-001. The phase 1/2 study, called IGNITE DMD, is designed to assess safety and efficacy of a single dose of SGT-001 in children and adolescents with DMD.