2024 Annual Meeting | SS10 - AAN Section Spotlight: Gene Therapies in Human Clinical Trials for CNS Diseases

Date Tuesday 04/16/24

Time 03:00 PM - 04:00 PM MDT Add To Calendar

Location Colorado Convention Center | 111

Session Format This program will be presented in-person only

On Demand This program is not expected to be available in the meeting's On Demand product.

Event Type Other

Director(s) Sonika Agarwal, MD, FAAN, Samuel A. Frank, MD, FAAN, Donald Gilbert, MD, FAAN, Deepa S. Rajan, MD, FAAN, Lauren Reoma, MD, FAAN

Topic(s) Child Neurology and Developmental Neurology, General Neurology

Learning Objectives Sponsored by the Child Neurology Section in collaboration with the Neurogenetics and Experimental Neurotherapeutics Sections. Participants should be able to describe basic mechanisms of gene therapies such as lentiviral vectors, adeno-associated viral vectors, and antisense oligonucleotides recently/currently in human clinical trials for application in central nervous system diseases; and discuss successes, failures, and treatments on the horizon for severe neurogenetic diseases in children and adults.

CME Available 0.00 CME credit

Core Competencies Patient Care & Procedural Skills, Practice-based Learning and Improvement, Systems-based Practice, Quality Improvement

Program Level Advanced

Recommended Audience Fellow, Resident, General Neurologist, Specialist Neurologist, Non-neurologist, Advanced Practice Provider, Neurohospitalist, Medical Student

Teaching Styles Case-based, Didactic, Audience Participation

Program Materials Program Evaluations

Event Timeline
03:00 PM - 04:00 PM MDT	Discussion	AAN Section Spotlight: Gene Therapies in Human Clinical Trials for CNS Diseases Samuel A. Frank, MD, FAAN, Donald Gilbert, MD, FAAN, Deepa S. Rajan, MD, FAAN

Faculty Disclosures
Sonika Agarwal, MD, FAAN	Dr. Agarwal has nothing to disclose.
Samuel A. Frank, MD, FAAN	Dr. Frank has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Frank has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sage Therapeutics. Dr. Frank has received personal compensation in the range of $500-$4,999 for serving as a Consultant for uniQure. Dr. Frank has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for AskBIO. Dr. Frank has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Frank has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Reata. Dr. Frank has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Teva. The institution of Dr. Frank has received research support from Huntington's Disease Society of America. The institution of Dr. Frank has received research support from Roche/Genentech. The institution of Dr. Frank has received research support from CHDI Foundation. The institution of Dr. Frank has received research support from Huntington Study Group. The institution of Dr. Frank has received research support from Cerevel.
Donald Gilbert, MD, FAAN	Dr. Gilbert has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Dr. Gilbert has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Illumina. Dr. Gilbert has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Emalex Biosciences. The institution of Dr. Gilbert has received research support from NIMH. The institution of Dr. Gilbert has received research support from Emalex Biosciences. The institution of Dr. Gilbert has received research support from PTC Therapeutics. The institution of Dr. Gilbert has received research support from Department of Defense. Dr. Gilbert has received publishing royalties from a publication relating to health care. Dr. Gilbert has received publishing royalties from a publication relating to health care. Dr. Gilbert has received personal compensation in the range of $0-$499 for serving as a Medical Second Opinion Expert with Teldoc/Advanced Medical. Dr. Gilbert has received personal compensation in the range of $10,000-$49,999 for serving as a Medical Expert with Department of Health and Human Services/Vaccine Injury Compensation Program.
Deepa S. Rajan, MD, FAAN	Dr. Rajan has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Medlink Neurology. The institution of Dr. Rajan has received research support from National Scleroderma Foundation. The institution of Dr. Rajan has received research support from Denali Therapeutics. The institution of Dr. Rajan has received research support from Ultragenyx . The institution of Dr. Rajan has received research support from Regenxbio. The institution of Dr. Rajan has received research support from Takeda. The institution of Dr. Rajan has received research support from Prevail Therapeutics. The institution of Dr. Rajan has received research support from Children's Neuroscience Institute. Dr. Rajan has received intellectual property interests from a discovery or technology relating to health care.
Lauren Reoma, MD, FAAN	Dr. Reoma has received research support from NIH. Dr. Reoma has a non-compensated relationship as a Vice Chair with AAN Experimental Neurotherapeutics Section that is relevant to AAN interests or activities. Dr. Reoma has a non-compensated relationship as a Federal Employee with NINDS/NIH that is relevant to AAN interests or activities. Dr. Reoma has a non-compensated relationship as a Member with ASENT Program Committee that is relevant to AAN interests or activities.