WebJEWELFISH. Research type. Research Study. Full title. AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, ... (SMA). RO7034067 has not been approved for marketing in any country.\nThe target sample size is up to 24 patients with Type 2 and Type 3 SMA (aged 12 to 60 years) ... Web9 nov. 2024 · Een aantal andere onderzoeken (zoals de Jewelfish en Rainbowfish trials) is nog niet afgerond. Wie komt voor Risdiplam in aanmerking en onder welke voorwaarden? Volwassen en kinderen van 2 maanden of ouder met SMA type 1 of 2 komen in het kader van dit programma alleen voor Risdiplam in aanmerking als Spinraza onvoldoende effect …
JEWELFISH: 24-month safety, pharmacodynamic and exploratory …
Web19 jul. 2024 · JEWELFISH, as of Dec. 1, 2024, had six patients with type 2 and six with type 3 disease, two SMA forms whose symptoms typically become evident in the first years of life. Their median age was 20, ranging from 13 to 52 years old. “So many of those patients are quite advanced,” Chiriboga said. http://pharmabiz.com/NewsDetails.aspx?aid=153816&sid=2 ilr technical
Evrysdi (risdiplam) for SMA SMA News Today
WebJEWELFISH is an open-label, noncomparative study of risdiplam in patients with SMA who were previously enrolled in Roche Study BP29420 (MOONFISH) with the splicing modifier RG7800 (RO6885247) (development discontinued) or previously treated with nusinersen, onasemnogene abeparvovec, or olesoxime (previous Roche acquired development … Web12 okt. 2024 · The JEWELFISH study enrolled the broadest and most diverse patient population ever studied in an SMA trial. Of the 174 people enrolled, 36% (n=63) were adults, 63% (n=105) had a Hammersmith... Web• JEWELFISH (NCT03032172)4 is an ongoing, multicentre, open-label study evaluating risdiplam in the broadest population ever studied in an SMA trial, including patients with Types 1–3 SMA with a wide range of ages (1–60 years) and disease severities, and who have previously received other DMTs. ilrs workshop