Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before six weeks of age, with most achieving motor milestones similar to children without SMAAll children were able to swallow and feed orally, with none requiring permanent ventilationEvrysdi is the only non-invasive SMA therapy and is approved in over 100 countries, with more than 16,000 people with SMA treated globally Basel, 14 October 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) presented positive two-year data from the ongoing RAINBOWFISH study at the 29th World Muscle Society (WMS) Congress, 8-12 October, 2024, assessing the efficacy and safety of Evrysdi® (risdiplam) in children with SMA who were treated pre-symptomatically as infants before six weeks of age (n=23).

积极的数据证实，Evrysdi在六周龄之前首次接受症状前治疗的儿童中具有有效性和安全性，大多数达到的运动里程碑与没有SMA的儿童相似。所有儿童都能够吞咽和口服喂养，不需要永久通气。Vrysdi是唯一一种非侵入性SMA疗法，已在100多个国家获得批准，2024年10月14日，全球有16000多名SMA患者接受了治疗。罗氏公司（six:RO，ROG；OTCQX:RHBY）在2024年10月8日至12日举行的第29届世界肌肉学会（WMS）大会上提供了持续两年的积极数据，评估了Evrysdi的有效性和安全性®（risdiplam）治疗SMA患儿，这些患儿在六周龄之前接受了婴儿症状治疗（n=23）。

The study found the majority of children achieved key motor milestones, were able to swallow and feed orally, and demonstrated cognitive skills typical of children without SMA, with none requiring permanent ventilation. “In children with SMA, motor neuron degeneration starts before the onset of symptoms, so time is of the essence if we hope to preserve muscle function,” said Laurent Servais, M.D., Ph.D., Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre.

该研究发现，大多数儿童达到了关键的运动里程碑，能够吞咽和进食，并且表现出没有SMA的儿童典型的认知技能，没有一个需要永久通气。。

“It’s heartening to see that through early intervention with Evrysdi these children have achieved important milestones like sitting, standing and walking that would typically be unattainable without treatment.” All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%) milestones as assessed by Bayley Scales of Infant and Toddler Development, third edition (BSID-III) and Hammersmith Infant Neurological Examination, Module 2 (HINE-2), with most achieving these mi.

“令人鼓舞的是，通过对Evrysdi的早期干预，这些儿童已经取得了重要的里程碑，如坐着，站立和行走，如果不进行治疗，这些里程碑通常是无法实现的。”所有接受Evrysdi治疗的儿童都有三个或更多SMN2拷贝（n=18），通过Bayley婴幼儿发育量表第三版（BSID-III）和Hammersmith婴儿神经系统检查模块2（HINE-2）评估，达到了站立和行走（100%）的里程碑，其中大多数达到了这些mi。

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