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This website is a non-promotional global resource intended to facilitate transparent scientific exchange regarding developments in medical research, diagnostics, and disease management. This global website is intended for healthcare professionals outside the UK, US, Canada, and Australia. The content on this website may include scientific information about experimental or investigational compounds, indications, and services that are not approved or valid in your country. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned on this website. Information available on this website does not constitute professional medical advice, and Roche and Genentech accept no responsibility for access to or use of the same.
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Congresses & Events
21 March, 2025
Congresses & Events
20 March, 2025
Congresses & Events
18 March, 2025
Congresses & Events
16 March, 2025
Congresses & Events
16 March, 2025
SUNFISH was a multicenter, two-part, randomized, placebo-controlled, double-blind clinical trial of risdiplam▼ in a broad and heterogeneous population with Types 2 and 3 SMA (aged 2–25 years at enrollment). This is the first presentation of motor function and safety data at the end of the 5-year SUNFISH study. Children, teenagers and adults with SMA showed improvement or stabilization in motor function outcomes, which is in contrast to the decline observed in an untreated population.
Congresses & Events
16 March, 2025
We report on a multicenter study assessing the feasibility, robustness, and sensitivity of using stride velocity 95th percentile (SV95C) to evaluate ambulatory function in patients with DMD under four years of age. Syde provided reliable, stride-level data, with SV95C distinguishing patients with DMD from age-matched controls. Early findings suggest that SV95C improves after steroid initiation and may capture early disease progression. Good adherence and high reliability support the use of digital biomarkers in this young population.
Congresses & Events
16 March, 2025
Adults currently account for ~50% of the SMA population worldwide. This study investigated motor function outcomes in adults with SMA treated with risdiplam▼ in the real world. These analyses used data from individuals enrolled in the PNCR network registry.
Congresses & Events
16 March, 2025
We report pooled cardiac safety outcomes from delandistrogene moxeparvovec studies with 1–5 years of follow-up in patients with Duchenne muscular dystrophy (DMD). Among 156 patients, two cases of myocarditis were resolved within three weeks, and on case of immune-mediated myositis stabilized within two weeks of treatment. Troponin I fluctuations were compatible with fluctuations observed in DMD. Long-term echocardiographic data showed stable left ventricular ejection fraction (LVEF), with no persistent cardiac injury. These findings indicate a manageable cardiac safety profile for delandistrogene moxeparvovec in this population of patients with DMD.
Congresses & Events
16 March, 2025
We report 3-year functional outcomes of ambulatory patients (aged 4.0–8.9 years) with Duchenne muscular dystrophy treated with delandistrogene moxeparvovec, compared to propensity-score-matched external controls (EC). Data from 50 treated patients across multiple studies were analyzed using a propensity-score-weighted median regression model and a mixed-effects model for repeated measures. Results showed clinically meaningful stabilization of disease over 3 years in the treatment group versus the ECs, with smaller decline in the North Star Ambulatory Assessment scores and improved performance in timed function tests. These findings support the long-term stabilization or slowing of disease progression with delandistrogene moxeparvovec compared to ECs.
Congresses & Events
16 March, 2025
We report 2-year functional and safety outcomes and Week 64 biological outcomes from patients treated with delandistrogene moxeparvovec in Part 1 of EMBARK. Results indicate stabilization or slowing of disease progression compared with well-matched external controls in functional outcomes prognostic for delaying loss of ambulation, with nominally statistically significant differences between the treatment group and external control cohort. Sustained micro‑dystrophin expression and localization to the sarcolemma was observed up to Week 64, and safety was consistent with prior experience.
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