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A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)

Not Yet RecruitingN/A

Sponsor: Hoffmann-La Roche

Conditions: Spinal Muscle Atrophy

This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with Type I and Type II Spinal Muscle Atrophy over 24 months of treatment. The study will allow retrospective collection of medical history data and previous participants visits from the medical records.

Eligibility overview

Sex: ALL

Age: — to 5 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria

Inclusion Criteria:

* Maximum age to be 6 months for type I participants and 5 years for type II participants at the time of diagnosis.
* Participants must start/have started treatment with Risdiplam within 6 months of diagnosis.
* Participant to be receiving adequate nutrition and hydration at index date, in the opinion of the Investigator.
* Participant to be adequately recovered from any acute illness at the time index date.

Exclusion Criteria:

* Participants not receiving treatment for disease with Risdiplam according to standard of care and in line with the current summary of product characteristics (SPC)/local labeling.
* Participants not receiving the Roche studied medicinal product, but a biosimilar.
* Concomitant or previous administration in any investigational drug or device study.
* Concomitant or previous administration of approved treatment for SMA other than Risdiplam.
* Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system diseases at index date.
* Participants requiring invasive ventilation or tracheostomy at index date.
* Participants requiring awake non-invasive ventilation due to respiratory insufficiency or with awake hypoxemia with or without ventilator support or with history of respiratory failure or severe pneumonia, and have not fully recovered their pulmonary function at the time of index date.
* Severe contractures of joints of lower limbs and upper limbs at the time of index date.