Help us write a new chapter in spinal muscular atrophy research.
Help us write a new chapter in spinal muscular atrophy research.
The story begins by joining the SAPPHIRE clinical study.
Consider participating in the SAPPHIRE study.
This clinical research study will look at an investigational drug called apitegromab (SRK-015) for people ages 2 to 21 with later-onset* spinal muscular atrophy (SMA) who are currently taking Spinraza® (nusinersen) or Evrsydi® (risdiplam).
* “Later-onset” SMA means you or your child has either Type 2 SMA (typically diagnosed before the age of 2 years) or Type 3 SMA (typically diagnosed after the age of 3 years). For more information about enrollment requirements, click here.
Travel expenses will be reimbursed and study participants will not be charged for any study doctor visits, laboratory work, tests, or procedures needed for the SAPPHIRE study.
The investigational drug (apitegromab) has not been approved by any health authorities, such as the US Food and Drug Administration (FDA), the European Medicine Agency (EMA), or the Medicines and Healthcare products Regulatory Agency (MHRA).