Web13 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 Functional Motor Scale Expanded (HFMSE) score of less than 10 at baseline, meaning their disease was very severe, and 83% (n=139) had … Web15 jun. 2024 · Among the five abstracts featured, new data includes 12-month safety, pharmacodynamic and interim exploratory efficacy data from the JEWELFISH study of EVRYSDI in people previously treated with SMA-targeting therapies across a broad range of ages (1–60 years), SMA types (1–3) and SMN2 copy number (1-5).
New Genentech Data for Evrysdi (risdiplam) Show ... - Business Wire
Web13 jun. 2024 · Enrolment for the JEWELFISH study, assessing safety and pharmacodynamic data in previously treated patients with SMA, who are now receiving risdiplam, is complete (n=174). Among the patients who completed 12 months of treatment with risdiplam, a median two-fold increase in SMN protein versus baseline was observed … home goods buffalo ny
Risdiplam on Spinal Muscular Atrophy - Clinical Trials Registry
Webhave been included in SUNFISH and JEWELFISH studies. In SUNFISH trial, 10 such patients have been included in the active arm and 8 in the pla cebo arm, and the following table recapitulates the respective observations after 12 m of treatment. As for JEWELFISH efficacy observations are not yet Web26 okt. 2024 · It was conducted in five study sites in South Africa, Uganda, Zambia and India. 1204 children aged under 16 years participated in the trial, including 127 children … Web11 jun. 2024 · Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated with another SMA-targeting therapy, including nusinersen and onasemnogene abeparvovec, showed the safety profile of Evrysdi and increase in SMN protein levels are consistent … hilton methodist church