A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam in adult and pediatric patients with spinal muscular atrophy

You may be able to join if

• 1. Males and females 6 months to 60 years of age inclusive (at screening)
• 2. Confirmed diagnosis of 5q-autosomal recessive SMA
• 3. Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previous treatment with nusinersen (defined as having received ≥ 4 doses of nusinersen, provided that the last dose was received ≥ 90 days prior to screening),olesoxime (provided that the last dose was received ≤ 18 months and ≥ 90 days prior to screening) or onasemnogene abeparvovec (AVXS-101) (provided that the time of treatment was ≥ 12 months prior to screening)
• 4. Adequately recovered from any acute illness at the time of screening and considered well enough to participate in the opinion of the Investigator
• 5. For patients aged 2 years or younger at screening: • Receiving adequate nutrition and hydration (with or without gastrostomy) at the time of screening, in the opinion of the Investigator • Medical care meets local accepted standard of care, in the opinion of the Investigator • Would be able to complete all study procedures, measurements and visits, and the parent or caregiver of the patient has adequately supportive psychosocial circumstances, in the opinion of the Investigator • Parent or caregiver of patient is willing to consider nasogastric, nasojejunal or gastrostomy tube placement, as recommended by the Investigator, during the study to maintain safe hydration, nutrition and treatment delivery • Parent or caregiver of patient is willing to consider the use of noninvasive ventilation, as recommended by the Investigator during the study