Researchers are looking for people with myasthenia gravis (MG) to participate in a multinational phase 2A clinical trial to evaluate the safety, tolerability and effectiveness of the investigational drug UCB7665. UCB7665, under development by UCB Pharma, is administered via subcutaneous (under-the-skin) injection and is designed to reduce the severity of muscle weakness and fatigue in . . .
With a focus on respiratory care in Duchenne muscular dystrophy (DMD), Santhera Pharmaceuticals has made available new resources for individuals with the disease. In addition, the company is seeking individuals to participate in a phase 3 clinical trial to test a respiratory drug in people with DMD. SIDEROS trial seeks participants Researchers are looking for . . .
MDA has awarded a human clinical trial grant totaling $750,000 to ALS ONE to explore the potential for a type of imaging called positron emission tomography (PET) to measure inflammation in the brain that could serve as a biomarker for ALS. ALS ONE is an alliance between four institutional leaders in ALS treatment development: Massachusetts . . .
Researchers are looking for children and adults with congenital myasthenic syndrome (CMS) to participate in a phase 3 study being conducted by Catalyst Pharmaceuticals to test the experimental drug amifampridine phosphate (brand name Firdapse).
Acceleron Pharma announced encouraging preliminary results from its phase 2 clinical trial to test the experimental drug ACE-083 in FSHD.
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.
Catalyst Pharmaceuticals has reported encouraging results from its second phase 3 clinical trial to test the investigational drug amifampridine phosphate (brand name Firdapse) in people with LEMS.
Cytokinetics has reported negative results from its international phase 3 VITALITY-ALS trial to test the investigational drug tirasemtiv in people with ALS.
