Web11 apr. 2024 · Rozpoczynamy zapisy na XI Weekend ze SMA-kiem. Rejestracji można dokonać na dwie formuły: biorąc udział na żywo lub online. Шановні СМА-родини, які не є громадянами Польщі та хотіли б взяти участь у нашій конференції – Ласкаво просимо! 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...
JEWELFISH - Health Research Authority
WebBackground: Nusinersen is approved for the treatment of 5q spinal muscular atrophy of all types and stages in patients of all ages. Although clinical trials have shown improvements in motor function in infants and children treated with the drug, data for adults are scarce. WebThe first study being presented was JEWELFISH, a trial evaluating the safety, tolerability and efficacy of risdiplam in people living with SMA Types 1-3, aged 1 to 60 years who had been previously treated with another SMA-targeting therapy, including nusinersen (Spinraza™) and onasemnogene abeparvovec (Zolgensma™). idmr soundcloud
P.110 JEWELFISH: 24-month safety and pharmacodynamic data in …
Web3 mei 2024 · Conclusions: JEWELFISH is ongoing at sites across Europe and the US, and will provide important data on the safety, PD and exploratory efficacy of risdiplam in a broad population of non-naïve patients with SMA. Disclosure: Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentec. Dr. Web12 jun. 2024 · Enrollment 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 … Web3 okt. 2024 · JEWELFISH (NCT03032172) is a multicentre, open-label study primarily evaluating the safety and tolerability of once-daily oral administration of risdiplam (RG7916) in patients aged 12–60 years with Type 2 or 3 SMA who have previously participated in a study with therapy targeting SMN2 splicing. Primary endpoints for Part 1 are safety, … idmr membership