Our science is just one way we champion patients. We believe in setting up a support system for patients and families that responds to not only their physical needs, but also their mental and emotional needs for well-rounded health. From raising disease awareness to funding research, our network uncovers hope.
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Spinal Muscular Atrophy
In partnership with Gilead Sciences
“We focus on the patients here; they’re the reason we’re at Scholar Rock.”
Senior Director, IT
We focus on the discovery and development of safe and effective therapies to find optimal solutions for those who need it.
How it Works
A clinical trial is a type of research involving human volunteers (also called participants) that follow strict protocols to evaluate whether a new drug is safe and effective in people. Clinical trials are usually conducted in multiple phases, each of which are designed to answer specific scientific questions. Data from these studies are then submitted to regulatory agencies, (e.g. US Food & Drug Administration), to gain approval of the new drug or treatment.
Find the safest dose
Test for beneficial effects and undesirable effects
Monitor adverse reactions
Test long term safety, in a diverse patient population
Focus on the study drug’s safety and tolerability. Phase 1 trials evaluate multiple doses in a small number of individuals that are, often, healthy volunteers.
Focus on the study drug’s safety and efficacy in patients with the disease or condition being studied. If a study drug is found to be reasonably safe in Phase 1 trials, it will then be investigated in Phase 2.
Focus on the study drug’s safety and efficacy, oftentimes compared to standard-of-care or control groups. Phase 3 trials typically evaluate much larger numbers of patients and follow the most rigorous protocols of the different phases. A study drug that passes Phase 3 can be submitted to regulatory agencies for approval to be marketed to the general public.
Pursue ongoing evaluation and monitor the study drug in patients, even after receiving regulatory approval.
Expanded Access Policy
As we advance our investigational therapies through clinical development, our goal is to provide access to these therapies at the appropriate time and in the correct manner for patients.
April 10, 2021
Scholar Rock Presents Biomarker Strategy to Support the SRK-181 DRAGON Phase 1 Proof-of-Concept Trial at the American Association for Cancer Research Annual Meeting
April 6, 2021
Scholar Rock Announces Positive 12-Month Top-Line Results From the TOPAZ Phase 2 Clinical Trial Evaluating Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
March 22, 2021
Scholar Rock Announces Publication of Preclinical Pharmacology, Pharmacokinetics, and Safety Profile of SRK-181 in the International Journal of Toxicology