UBC Up for Clinical Research Team of the Year
The Citeline Awards by Informa Pharma Intelligence have named UBC's Rare Disease Gene Therapy Registry Team as a finalist for Clinical Research Team of the Year.
BLUE BELL, Pa., Nov. 18, 2021 /PRNewswire/ -- The Citeline Awards celebrate achievements within biopharmaceutical research and development. They bring together leaders from biopharmaceutical, CRO, and diagnostic industries to recognize the best work from early and late-stage research, to advances in technological tools, to innovations in trial design and execution.
UBC's Rare Disease Gene Therapy Registry Team has been shortlisted after undertaking a highly successful collaboration in the conduct of a prospective observational registry of patients with Spinal Muscular Atrophy (SMA). The program successfully enrolled approximately 300 patients into the global registry within an initial two-year period, which produced robust data used to meet regulatory objectives, inform SMA experts, and support publications. This data has ultimately contributed to more thoroughly understanding the efficacy and safety of novel therapies for SMA.
SMA is a neurogenetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle). SMA most often affects babies and children and makes it hard for them to use their muscles, and children can have trouble controlling head movement, sitting without help, and even walking. In some cases, they can have trouble swallowing and breathing as the disease gets worse. The main objective of this registry is to assess the long-term outcomes of patients with a diagnosis of SMA in the context of advances in treatment options.
SMA used to be one of the most common genetic causes of infant mortality. New disease-modifying treatments have changed the disease trajectories and the best results are seen if treatment is initiated in the pre-symptomatic phase of the disease. Systematic real-world data collection and additional clinical trials are needed to fill evidence gaps concerning safety and efficacy. The data that has and will be gathered in this registry is ambitiously addressing evidence needs for the SMA community at large.
The Gene Therapy Registry Team is a cross-functional team that has worked daily for over two years to propel the registry forward. This includes work in engaging the SMA community, effectively onboarding global institutions, driving data-sharing partnerships, and disseminating the knowledge gained from the data. This well-earned recognition highlights the important efforts that go on to effect change against rare diseases.
About UBC
United BioSource LLC (UBC) is a leader in the biopharmaceutical market which provides integrated clinical, safety, and commercialization services. UBC brings together renowned scientific research and operations experts with innovative technologies, allowing for the best patient and healthcare provider experience. Comprehensive, end-to-end services cover product and patient population characterization during development and market entry, as well as a focus on the patient experience, safety, and adherence. For additional information, visit https://ubc.com/.
SOURCE United BioSource Corporation
Share this article