Combined Treatments for Spinal Muscular Atrophy

Treatments that could be combined with SMN-directed therapies, example Spinraza  with other new drugs. The research is ready, Roche is doing it in the JEWELFISH Study. This could be a big new step in the future Hope in treatments that could be combined with SMN-directed therapies; for example, drugs that act… Att fortsätt läsa

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… Att fortsätt läsa

PTC Therapeutics Announces Positive Preliminary Clinical Data from the FIREFISH Study in Type 1 SMA Patients

The clinical data were presented at the annual meeting of the American Academy of Neurology. The presentation from Part 1, the Dose Finding section, of the ongoing FIREFISH study. The open-label study investigated RG7916, an oral survival motoneuron 2 (SMN2) splice modifier, in type 1 SMA patients. The data from… Att fortsätt läsa

PTC Therapeutics, gibt positive Vorläufige Klinische Daten der FIREFISH-Studie bei Typ-1-SMA-Patienten bekannt

die klinische Daten wurden auf der Jahrestagung der American Academy of Neurology vorgestellt. Die Präsentation aus Teil 1, dem Dosisfindungs-Teil, der laufenden FIREFISH-Studie bekannt. Die Open-Label-Studie untersucht RG7916, ein orales Survival Motoneuron 2 (SMN2) Spleißmodifikator, bei Typ-1-SMA-Patienten. Die Daten aus Teil 1 wurden auf der Jahrestagung der American Academy of… Att fortsätt läsa