Risdiplam is an investigational oral therapeutic for the treatment of spinal muscular atrophy (SMA). SMA is caused by reduced levels of SMN protein due to deletion or mutation in the SMN1 gene and low SMN production from a related SMN2 gene due to alternative splicing. Risdiplam modifies the splicing of SMN2 pre-mRNA and increases the production of survival motor neuron (SMN) protein, which is critical for the proper function of motor neurons as well as peripheral organs and tissues such as muscle, liver, bone and vasculature. 

Risdiplam is being investigated in three ongoing clinical studies designed to evaluate the safety, efficacy, and tolerability of risdiplam in patients with SMA. The studies include:

  • FIREFISH (NCT02913482) in infants with type 1 SMA
  • SUNFISH (NCT02908685) in Type 2 and 3 SMA patients
  • JEWELFISH (NCT03032172) in Type 2 and 3 SMA patients previously treated with SMN2 splicing modifiers

All three studies are recruiting eligible SMA patients globally. More information on each trial is available by visiting https://clinicaltrials.gov and searching with the trial’s identification number.

The Risdiplam SMA program is a three-party collaboration between Roche, PTC Therapeutics, and the SMA Foundation.  Roche is leading clinical development and the program is overseen by a joint steering committee comprised of collaboration partners.

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