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Presentation Description
The neurofibromatoses include distinct clinical and genetic conditions including Neurofibromatosis Type 1 (NF1), NF2-schwannomatosis (NF2-SWN), LZTR1-schwannomatosis (LZTR1-SWN), SMARCB1-schwannomatosis (SMARCB1-SWN) and other chromosome 22-related schwannomatoses.
Within each of these conditions are a range of important and impactful manifestations that unfold over a person’s lifetime. The genetic, molecular, and clinical complexity underlying these conditions requires a systems and teams-based approach to understanding the pathophysiology and developing effective therapies.
Through initiatives within the Neurofibromatosis Therapeutic Acceleration Program (NTAP) and Synodos for NF2 stakeholders, including people living with the condition, advocates, discovery, translational and clinical scientists, regulatory experts, and commercial partners have worked to accelerate the pace of meaningful therapeutic discovery for key manifestations of these conditions.
Dr Blakeley will share the philosophy underlying team science and vignettes of recent successes within NF1 and NF2-SWN. She will also highlight areas of need for further growth and investment and ongoing strategies to expand the breadth and depth of scientific and clinical progress via NTAP.
Within each of these conditions are a range of important and impactful manifestations that unfold over a person’s lifetime. The genetic, molecular, and clinical complexity underlying these conditions requires a systems and teams-based approach to understanding the pathophysiology and developing effective therapies.
Through initiatives within the Neurofibromatosis Therapeutic Acceleration Program (NTAP) and Synodos for NF2 stakeholders, including people living with the condition, advocates, discovery, translational and clinical scientists, regulatory experts, and commercial partners have worked to accelerate the pace of meaningful therapeutic discovery for key manifestations of these conditions.
Dr Blakeley will share the philosophy underlying team science and vignettes of recent successes within NF1 and NF2-SWN. She will also highlight areas of need for further growth and investment and ongoing strategies to expand the breadth and depth of scientific and clinical progress via NTAP.