Amy 9 Jahre jung wurde mit der spinale Muskelatrophie geboren, durch den Gendefekt verliert Amy ihre Muskelkraft Ihre Familie sammelt nun Geld für ein Auto welches auf Amy speziell zugeschnitten ist. Denn mit der spinale Muskelatrophie ist Amy abhängig von ihrem Rollstuhl und den vielen Hilfsmittel wie eine mobile Beatmung. Damit das Mädchen nicht immer […]
Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies with Risdiplam
risdiplam- RG7916 is well tolerated at all dose levels with no drug-related safety findings – SOUTH PLAINFIELD, N.J., June 16, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy […]
The Drug Development Process > Phase II clinical trials with Reldesemtiv in Patients with Spinal Muscular Atrophy (SMA II-III)
presented in an oral presentation by John W. Day, MD, Ph.D., Professor of Neurology and Paediatrics (Genetics), Stanford University. This hypothesis-generating study met its primary goal. It fulfills the effect and safety profile. In collaboration with Astellas, Cytokinetics is developing reldesemtiv as a potential treatment for people with SMA and certain other debilitating diseases and […]