Understanding the ins and outs of a rare disease like atypical hemolytic uremic syndrome (aHUS) is a difficult task for ...
A classification of hemolytic uremic syndrome and thrombotic thrombocytopenic purpura and related disorders. Kidney Int. 70 , 423–431 (2006). Article CAS Google Scholar ...
Alport syndrome, and atypical hemolytic uremic syndrome." The results revealed that 10 of the 90 patients (11% of the final ...
Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. | Solid Biosciences has ...
Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...
Atypical hemolytic uremic syndrome is an ultra-rare genetic disease that causes anemia and damages kidney function. There is an approved treatment — a therapeutic antibody called eculizumab ...
Solid Biosciences (SLDB) announced initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy ...
Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.
They are thus reported to be useful for the treatment of age-related macular degeneration, schizophrenia, atypical hemolytic uremic syndrome, membranous nephropathy, myasthenia gravis, paroxysmal ...
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