Sma-europe.eu Website Review

sma europe campaigns to improve the quality of life of people who live with sma, to bring effective therapies to patients in a timely and sma europe campaigns to improve the quality of life of people who live with sma, to bring effective therapies to patients in a timely and roche’s risdiplam has been ganted prime designation by the european medicines agency (ema) for treatment of spinal muscular atrophy (sma) risdiplam has the potential to be the first oral medicine for the treatment of sma types 1, 2 and 3; european medicines agency prime. scholar rock announces positive opinion by the ema on orphan drug designation for srk-015 for the treatment of sma. scholar rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the european medicines agency’s (ema’s) committee for orphan medicinal products (comp). combinatorial aso therapy using smn-dependent and smn-independent protection against sma. university of cologne, was granted an sma europe award to see if combining a compound which increases smn production (such as spinraza™), with a molecule involved in endocytosis called neurocalcin delta (ncald), will counteract the disturbed function of motor neurons, even in the most severely affected type of sma. the nerve cells involved in muscle function. the severity of sma is determined by the copy number of a second gene, smn2, which also produces the smn protein, although only about 10% of the protein produced is viable. recent studies suggest that there are additional cellular mechanisms which determine the severity of disease, in addition to copy numbers of smn2. despite advanced understanding of the genetics involved in sma, the cellular mechanisms which cause sma are not yet completely understood. the investigator’s team previously observed that a mechanism called endocytosis, the process which allows cells to recycle and transport molecules, is impaired in sma, as a consequence of low smn levels. this process is very important in nerve cells,