2020 Update on Gene Therapy for CNS Diseases Explore the latest advancements in gene therapy for CNS diseases with researcher Jim Wilson. Learn about challenges, clinical trials, and future directions in this comprehensive update.
Background on Neuren Pharmaceuticals and NNZ 2591 Explore Neuren Pharmaceuticals’ progress in developing drugs for neurodevelopmental disorders. Learn about their lead program, NNZ-2591, and its promising results in treating synaptic plasticity deficits.
Patient’s Role in Drug Development Explore David Meeker’s insights on the crucial role patients play in drug development for rare diseases, emphasizing the power of storytelling and community resilience.
Putting Patients at the Center: An Overview of Taysha Explore Taysha Gene Therapies’ mission to treat monogenic diseases of the central nervous system. Learn about their patient-centric approach, innovative gene therapy development, and commitment to collaboration.
Validation of ORCA Outcome Measure Explore Bryce Reeve’s article on the validation of the ORCA measure, a tool developed to assess communication abilities in children with Angelman Syndrome. Discover its development, testing, and implementation.
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