What is the purpose of our JEWELFISH clinical trial?

The JEWELFISH trial examines risdiplam in people between 6 months – 60 years of age with any Type of SMA.

The clinical trial is for people who have previously participated in the Moonfish study (BP29420) or who received previously treatment with olesoxime or nusinersen.

The investigational treatment is taken once daily in liquid form by mouth or feeding tube.

How many patients will be enrolled?

The clinical trial will include approximately 125 patients.

How long is treatment given for?

The clinical trial runs for 24 months of treatment after which participants will be offered to continue treatment as part of an extension phase of the study. After stopping treatment participants will be followed for a 12 months follow up period. All patients will receive risdiplam. There is no placebo in this clinical trial.

What are the eligibility criteria to take part?

1. Be between 6 months and 60 years of age at enrollment
2. Be medically diagnosed with SMA
3. Have previously participated in the Moonfish study Or received previously treatment with nusinersen (≥4 doses of nusinersen, provided that the last dose was received ≥90 days prior to screening) or olesoxime (provided that the last dose was ≤12 months and ≥90 days prior to screening)
4. Able to travel to the study site several times during the study period
5. Not currently enrolled in another clinical trial
6. Have not received gene or cell therapy
7. Be able to follow study procedures
8. Be able to take a daily oral medication either by mouth or by feeding tube
9. Have not had an eye disease within the past year
10. Be able to allow eye examinations to be performed
Please refer to for more inclusion and exclusion criteria.

I am interested in joining JEWELFISH – what should I do?

Please contact your treating physician to discuss the clincal trial.



Your local patient group may also be able to provide you with additional information.
You can also contact us if you need more information.

Where is my nearest study centre?

Locate hospitals that are participating in the JEWELFISH trial using our Find My Trial tool.

My country is not listed in and is not found using FIND MY TRIAL. How can I take part in JEWELFISH?

Participating in a trial in another country will be at the discretion of the investigator and is decided on an individual basis.

We can put you in contact with people who can answer your questions about Roche clinical trials in another country.

You can also regularly check back at and Find My Trial for newly opened sites.

Could I be given a placebo if I take part?

No, the JEWELFISH trial is an open-label study, so all patients will be given risdiplam.

Who will pay the costs associated with participation?

Roche will cover the cost of study medication, visits, assessments and reasonable costs of travel.