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Hospital for Special Care’s Neuromuscular Clinical Trials team has dramatically expanded access to clinical trials to patients with ALS, muscular dystrophy and Parkinson’s disease — neuromuscular diseases that can have a profound impact on mobility and other core functions including speech and respiration.
What have been the team’s significant contributions to the organization in the past year?
We have been selected as the only HEALEY ALS Platform Clinical Trial Site in Connecticut and one of only three U.S. Food and Drug Administration expanded access program sites for CNM-AU8 in the country. We provide extensive outreach and support to patients, help them navigate the complex eligibility criteria of clinical trials, and to understand the potential risks and benefits of participation.
We have been highly effective advocates, securing resources to expand access to programs that make experimental drug regimens accessible to patients who do not meet eligibility criteria for the Healey trials but may experience improvements in quality and length of life.
What are some major challenges the team had to overcome?
We designed and implemented extensive safety protocols to allow immunocompromised patients to continue their clinical trials throughout the pandemic.
Patients, particularly patients with ALS, simply cannot afford lost time when facing a terminal illness. Our efforts have enhanced and prolonged the lives of mothers and fathers, business and community leaders.
What’s the team’s next major goal?
Our primary goal is to help advance the search for effective treatments or cures for neuromuscular diseases, including ALS. Expanding access to clinical trials for patients through collaboration with our regional partners is essential to our patients and their families.
Other Team Members: Katharine Coggshall, Clinical Research Support; Natalie Schmitt-Cartwright, Clinical Research Coordinator; Zanib Iqbal, Clinical Research Coordinator; Stephanie Beck, Outcomes Measures
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