The most up-to-date information in lay terms on the progress of all of the AHCF and Collaborative funded research projects!
Dr. Al George – Northwestern University Will present an update on promising findings from both the AAV and ASO projects in his lab:
The first is relevant to the efficacy of the vector for the AAV project. Dr. George’s lab found that the vector was successful in cultured neurons.
The second finding is relevant to the use of ASOs as an effective therapy. This finding reflects the significant potential of ASOs to treat AHC.
Dr. Nutan Sharma & Dr. Laurie Ozelius – Massachusetts General/Harvard
Longitudinal Natural History presentation – what phase of development is it in, and what is next?
Research Clinic – Thursday all day by appointment. Old families update their records, new families become part of the AHC Clinical registry and biobank.
Nina Frost, BA, MPhil– Hope For Annabel President
Update on currently funded research projects, Jax labs, Dr. Liu-Harvard, Dr. Clapcote – England
Introduce CZ grant
Development of new research projects
Investigation into new research areas and describe research infrastructure
Dr. Matthew Campbell– Ireland
20% without mutation – intro to lab
Dr Natalia Morsci
Animal Models – will provide families an introduction to AHC animal models (more than a mouse)
Medical – AHC experts weigh in!
Physician’s panel – Saturday – Led by Dr. Kathryn Swoboda, including Dr. Marcio Sotero, Dr. Ken Silver, Dr. Al George, Dr. Hendrik Rosewich, Dr. Nutan Sharma, Dr. Sho Yano, Dr. Terry Sanger – Parents Q & A on AHC-related questions including Dystonia, Genetics, and Epilepsy.
Office Hours – Thursday – Open to all AHC families, caregivers, and physicians, we will be holding designated informal group discussions with various experts on Dystonia, Genetics, Neurology, and Epilepsy.
Sarah Spurling – MS Ph.D. LP HSPP “Navigating the psychosocial impacts of Chronic illness”.