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Articles by L. Mary Smith
06.14.21 -- Severe Disease Increases Necessity For A Patient-Centric Approach
Newsletter | June 14, 2021 By Ed Miseta, chief editor, Clinical Leader Spinal muscular atrophy (SMA) is a disease caused by a genetic deficiency in a gene known as SMN1, which leads to patients being unable to perform basic tasks such as standing or walking. Scholar Rock recently completed a Phase 2 proof-of-concept trial and is excited about the results and the potential it represents for patients with SMA. By L.
06.08.21 -- Best Practices In Clinical Trials For Patients With Rare Diseases
Newsletter | June 8, 2021 By L. Mary Smith, Ph.D., SpringWorks Therapeutics One way to improve on running your rare disease clinical trial is to foster collaborative partnerships with patient advocacy organizations and to stay connected to patients throughout the trial. Here's how to do that.
Best Practices For Designing And Running Clinical Trials For Patients With Rare Diseases
By L. Mary Smith, Ph.D., SpringWorks Therapeutics Most people agree that drug development is risky and challenging, and as an industry, we are constantly searching for ways to improve the drug development process – particularly in the rare disease space.
Best Practices For Designing And Running Clinical Trials For Patients With Rare Diseases
By L. Mary Smith, Ph.D., SpringWorks Therapeutics Most people agree that drug development is risky and challenging, and as an industry, we are constantly searching for ways to improve the drug development process – particularly in the rare disease space.
Best Practices For Designing And Running Clinical Trials For Patients With Rare Diseases
By L. Mary Smith, Ph.D., SpringWorks Therapeutics Most people agree that drug development is risky and challenging, and as an industry, we are constantly searching for ways to improve the drug development process – particularly in the rare disease space.
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