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2023 Annual Meeting | S14 - General Neurology: Emerging Therapies

Monday 04/24/23
01:00 PM - 03:00 PM EST Add To Calendar
This program will be presented both in-person and online
This program will be available in the meeting's On Demand product.
Scientific Platform Session
General Neurology
A group of abstracts covering a similar topic presented in an oral format. Presentations are eight minutes in length with four minutes for question and answer with the author.
2.00 CME credits
Fellow, Resident, General Neurologist, Specialist Neurologist, Non-neurologist, Advanced Practice Provider, Neurohospitalist, Medical Student
Didactic

Program Materials Program Evaluations

Event Timeline
01:00 PM - 01:12 PM EST Abstract 001 - NfL Levels Significantly Decrease in Response to Treatment with Patisiran or Vutrisiran in hATTR Amyloidosis with Polyneuropathy
Michael J. Polydefkis, MD, FAAN
01:12 PM - 01:24 PM EST Abstract 002 - Comparison of Therapeutic Efficacy of Gene Therapy for Tuberous Sclerosis Type 2 with Standard of Care Everolimus in Preclinical Model
Edwina Abou Haidar, MBBS, MS
01:24 PM - 01:36 PM EST Abstract 003 - Comparison of Efficacy Outcomes with Vutrisiran vs. Patisiran in hATTR Amyloidosis with Polyneuropathy: Post-hoc Analysis of the HELIOS-A Study
Michael J. Polydefkis, MD, FAAN
01:36 PM - 01:48 PM EST Abstract 004 - A Phase 1, First-in-human, Randomized, Double-blind, Placebo-controlled, Single and Multiple Ascending Intravenous Dose Study of a novel TREM2 agonist (VGL101) in Healthy Volunteers (HVs)
Andreas Meier, MD
01:48 PM - 02:00 PM EST Abstract 005 - Effect of Patisiran on Polyneuropathy and Cardiomyopathy in Patients with hATTR Amyloidosis with V122I/T60A Variants: A Phase 4 Observational Study
Raymond L. Comenzo
02:00 PM - 02:12 PM EST Abstract 006 - Enabling Subcutaneous Dosing of Gantenerumab in Alzheimer’s Disease
Janice W. Smith, PhD
02:12 PM - 02:24 PM EST Abstract 007 - Longer Milestone-Free Time in Patients With Amyotrophic Lateral Sclerosis Treated With IV Edaravone vs Not Treated With IV Edaravone: Results From a US Administrative Claims Analysis
Malgorzata Ciepielewska
02:24 PM - 02:36 PM EST Abstract 008 - Therapeutic Potential of Fosgonimeton, a Small-Molecule Positive Modulator of the Neurotrophic HGF/MET Pathway, in Neurodegenerative Conditions
Kevin Church, PhD
02:36 PM - 02:48 PM EST Abstract 009 - Angubindin-1 opens the blood–brain barrier in vivo for delivery of antisense oligonucleotide to the central nervous system
Hiroya Kuwahara, MD, PhD
02:48 PM - 03:00 PM EST Abstract 010 - Remote multimodal monitoring of motor neuron disease progression using wearable sensors and digital assessments
Ashkan Vaziri, PhD
Faculty Disclosures
Jeffrey Zhang Jeffrey Zhang has received personal compensation for serving as an employee of Princeton Pharmatech. Jeffrey Zhang has received stock or an ownership interest from CalciMedica.
Emre Aldinc Emre Aldinc has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Emre Aldinc has stock in Alnylam Pharmaceuticals.
Davide Pareyson, MD No disclosure on file
James Lim Mr. Lim has nothing to disclose.
Kevin Church, PhD Dr. Church has received personal compensation for serving as an employee of Athira Pharma. Dr. Church has stock in Athira Pharma. Dr. Church has received intellectual property interests from a discovery or technology relating to health care.
Jewel Johnston Dr. Johnston has received personal compensation for serving as an employee of Athira Pharma, Inc. Dr. Johnston has stock in Athira Pharma, Inc. An immediate family member of Dr. Johnston has stock in Athira Pharma, Inc. Dr. Johnston has received intellectual property interests from a discovery or technology relating to health care.
Saurabh Malhotra, MD Dr. Malhotra has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer Inc.. Dr. Malhotra has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Pfizer Inc. . Dr. Malhotra has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alnylam Therapeutics.
Agnès Portron, Other Mrs. Portron has nothing to disclose.
Xandra O. Breakefield, PhD Dr. Breakefield has nothing to disclose.
Ram Kinker Mishra, PhD Dr. Mishra has received personal compensation for serving as an employee of Biosensics.
Malgorzata Ciepielewska Malgorzata Ciepielewska has received personal compensation for serving as an employee of Mitsubishi Tanable Pharma America .
Francy Y. Shu, MD Dr. Shu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH. Dr. Shu has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alnylam. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for The CM Group. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for T&Associates. The institution of Dr. Shu has received research support from Biogen. The institution of Dr. Shu has received research support from Alnylam.
Ryan O'Mara, Other Mr. O'Mara has received personal compensation for serving as an employee of VIgil Neuroscience, LLC.
Zachary Simmons, MD, FAAN Dr. Simmons has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cytokinetics. The institution of Dr. Simmons has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amylyx. Dr. Simmons has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Simmons has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clene. Dr. Simmons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Simmons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Corcept. The institution of Dr. Simmons has received personal compensation in the range of $50,000-$99,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Wiley. The institution of Dr. Simmons has received research support from MGH. The institution of Dr. Simmons has received research support from MT Pharma. The institution of Dr. Simmons has received research support from University of Kansas. The institution of Dr. Simmons has received research support from ALS Association. The institution of Dr. Simmons has received research support from Clene.
Chafic Y. Karam, MD Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Karam has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neuroderm.
Jennifer Cramton Barnes, PhD Dr. Barnes has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Dr. Barnes has received personal compensation for serving as an employee of Acceleron Pharma Inc. Dr. Barnes has stock in Alnylam Pharmaceuticals. Dr. Barnes has stock in Acceleron Pharma Inc.
Andree-Anne Berthiaume, PhD Dr. Berthiaume has received personal compensation for serving as an employee of Athira Pharma. Dr. Berthiaume has stock in Athira Pharma. An immediate family member of Dr. Berthiaume has stock in Athira Pharma.
David Stiles, PhD Dr. Stiles has received personal compensation for serving as an employee of Vigil Neuroscience. Dr. Stiles has received personal compensation for serving as an employee of Vertex Pharmaceuticals.
Roberta Beauchamp, Other Ms. Beauchamp has nothing to disclose.
Michael J. Polydefkis, MD, FAAN Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharmaceutical . Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen-Idec. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer.
Sharay E. Setti, PhD Dr. Setti has received personal compensation for serving as an employee of Athira Pharma. Dr. Setti has stock in Athira Pharma.
Lin-Na Chou, Other Ms. Chou has received personal compensation for serving as an employee of Alnylam Pharmaceuticals, Inc..
David D. Adams David D. Adams has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ALNYLAM. David D. Adams has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer.
Joel Fernandez MD, MD Dr. Fernandez MD has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Zoll. The institution of Dr. Fernandez MD has received research support from Pfizer.
Sevda Lule, PhD Dr. Lule has received personal compensation for serving as an employee of Codiak Biosciences.
Hiroya Kuwahara, MD, PhD Dr. Kuwahara has nothing to disclose.
Casey Maguire, PhD Dr. Maguire has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Chameleon Biosciences. Dr. Maguire has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sphere Gene Therapeutics. Dr. Maguire has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Skylark Bio. Dr. Maguire has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sphere Gene Therapeutics. Dr. Maguire has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for CLS Therapeutics. Dr. Maguire has received stock or an ownership interest from Chameleon Biosciences. Dr. Maguire has received stock or an ownership interest from Sphere Gene Therapeutics. Dr. Maguire has received stock or an ownership interest from Skylark Bio. Dr. Maguire has received research support from SwanBio. Dr. Maguire has received research support from BridgeBio. Dr. Maguire has received research support from Wayvector Inc. Dr. Maguire has received intellectual property interests from a discovery or technology relating to health care.
Chongshu Chen Chongshu Chen has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Chongshu Chen has stock in Alnylam Pharmaceuticals.
Ying Liu Ying Liu has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for MPTA.
Kelly Neelon, PhD Dr. Neelon has received personal compensation for serving as an employee of BridgeBio. Dr. Neelon has received personal compensation for serving as an employee of Vigil NeuroScience. An immediate family member of Dr. Neelon has received personal compensation for serving as an employee of Crisper Therapeutics. An immediate family member of Dr. Neelon has received personal compensation for serving as an employee of Mana Therapeutics. Dr. Neelon has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for BridgeBio. Dr. Neelon has stock in BridgeBio. Dr. Neelon has stock in VigilNeuro Science. An immediate family member of Dr. Neelon has stock in Cripser Therapeutics. An immediate family member of Dr. Neelon has stock in Mana Therapeutics. Dr. Neelon has received intellectual property interests from a discovery or technology relating to health care.
Satoshi Zeniya, MD,PhD Dr. Zeniya has nothing to disclose.
Ronald Zolty, MD,PhD Dr. Zolty has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Zolty has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Zolty has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alnylam.
Elena Yureneva, MD Dr. Yureneva has received personal compensation for serving as an employee of Alnylam. Dr. Yureneva has stock in Alnylam.
Dietmar Schwab, PhD Dr. Schwab has received personal compensation for serving as an employee of F. Hoffmann-La Roche Ltd. Dr. Schwab has stock in F. Hoffmann-La Roche Ltd..
Frank Birklein, MD Dr. Birklein has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. The institution of Dr. Birklein has received research support from Alnylam.
Ashkan Vaziri, PhD Dr. Vaziri has received personal compensation for serving as an employee of Biosensics.
Dominik Lott Dominik Lott has received personal compensation for serving as an employee of Roche. Dominik Lott has received stock or an ownership interest from Idorsia Pharmaceuticals.
John Vest John Vest has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. John Vest has received stock or an ownership interest from Alnylam Pharmaceuticals. John Vest has received intellectual property interests from a discovery or technology relating to health care.
Jakub Wojtowicz, Other Mr. Wojtowicz has received personal compensation for serving as an employee of F. Hoffmann-La Roche. Mr. Wojtowicz has stock in F. Hoffmann-La Roche.
Vijaya Ramesh Vijaya Ramesh has nothing to disclose.
Paul Nioi Paul Nioi has received personal compensation for serving as an employee of Alnylam. Paul Nioi has received stock or an ownership interest from Alnylam.
Pike See Cheah, PhD The institution of Dr. CHEAH has received research support from MInistry of Higher Education, MALAYSIA.
Evan A. Thackaberry, PhD Dr. Thackaberry has received personal compensation for serving as an employee of Vigil Neuroscience. Dr. Thackaberry has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Samsara Biocapital. Dr. Thackaberry has stock in Vigil Neurosciences.
Jose Joaquin Casado, Other Mr. Casado has received personal compensation for serving as an employee of BioSensics LLC.
Shilpa Prabhakar, Sr., Other Miss Prabhakar has nothing to disclose.
Kelley Elizabeth Capocelli, MD Dr. Capocelli has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Dr. Capocelli has stock in Alnylam Pharmaceuticals.
Carsten Hofmann Carsten Hofmann has received personal compensation for serving as an employee of F. Hoffmann-La Roche Ltd. Carsten Hofmann has received stock or an ownership interest from F. Hoffmann-La Roche Ltd.
Remy Kohler Mr. Kohler has nothing to disclose.
Melissa Hagan, PhD Dr. Hagan has received personal compensation for serving as an employee of Mitsubishi Tanabe Pharma America.
Edwina Abou Haidar, MBBS, MS Dr. Abou Haidar has nothing to disclose.
Robert Taylor, PhD Dr. Taylor has received personal compensation for serving as an employee of Athira Pharma, Inc. Dr. Taylor has stock in Athira Pharma, Inc.. Dr. Taylor has received intellectual property interests from a discovery or technology relating to health care.
Raymond L. Comenzo Raymond L. Comenzo has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Raymond L. Comenzo has received intellectual property interests from a discovery or technology relating to health care. Raymond L. Comenzo has received intellectual property interests from a discovery or technology relating to health care.
Akiko Yoshinaga, MD Dr. Yoshinaga has nothing to disclose.
Andrew Geronimo The institution of Andrew Geronimo has received research support from BioSensics, LLC.
Frank Boess Frank Boess has received personal compensation for serving as an employee of F.Hoffmann-LaRoche. Frank Boess has stock in F. Hoffmann-La Roche. Frank Boess has received intellectual property interests from a discovery or technology relating to health care.
Yessar M. Hussain, MD The institution of Dr. Hussain has received research support from Alnylam Pharma.
David Danese, Other Mr. Danese has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. An immediate family member of Mr. Danese has received personal compensation for serving as an employee of Biogen. An immediate family member of Mr. Danese has received personal compensation for serving as an employee of Generate Biomedicines. Mr. Danese has stock in Alnylam Pharmaceuticals. An immediate family member of Mr. Danese has stock in Biogen.
Hans Nienhuis, MD,PhD The institution of Dr. Nienhuis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. The institution of Dr. Nienhuis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam.
Tetsuya Nagata, MD, PhD Dr. Nagata has nothing to disclose.
Beate Bittner Dr. Bittner has received personal compensation for serving as an employee of F. Hoffmann-La Roche Ltd.. Dr. Bittner has stock in F. Hoffmann-La Roche Ltd..
Senda Ajroud-Driss, MD, FAAN Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx Pharmaceuticals. Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Ajroud-Driss has received research support from ALSA. Dr. Ajroud-Driss has received publishing royalties from a publication relating to health care.
Urvi G. Desai, MD, FAAN Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Acceleron. Dr. Desai has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL Behring. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx.
Madeline Merkel Madeline Merkel has received personal compensation for serving as an employee of Alnylam Pharmaceuticals.
Sherif Reda, PhD Dr. Reda has received personal compensation for serving as an employee of Athira Pharma, Inc. . Dr. Reda has stock in Athira Pharma, Inc. .
Andrew James Marsh Mr. Marsh has received personal compensation for serving as an employee of Vigil Neuroscience, Inc.. Mr. Marsh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Vigil Neuroscience, Inc..
Yoshiki Sekijima, "MD, PhD" Yoshiki Sekijima, "MD, PhD" has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alnylam. Yoshiki Sekijima, "MD, PhD" has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Pfizer.
Marcia Waddington Cruz, MD Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sobi. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ionis. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NovoNordisk.
Spyridon Papapetropoulos, MD, PhD Dr. Papapetropoulos has received personal compensation for serving as an employee of Vigil Neuroscience . Dr. Papapetropoulos has received personal compensation for serving as an employee of Bionomics Ltd. Dr. Papapetropoulos has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for Lipocine Inc. Dr. Papapetropoulos has stock in Vigil Neuroscience. Dr. Papapetropoulos has stock in Lipocine. Dr. Papapetropoulos has stock in Bionomics Ltd.
Anat Stemmer-Rachamimov Anat Stemmer-Rachamimov has nothing to disclose.
Rajasimhan Rajagovindan, PhD Dr. Rajagovindan has received personal compensation for serving as an employee of Biogen. Dr. Rajagovindan has received personal compensation for serving as an employee of Vigil. Dr. Rajagovindan has stock in Biogen. Dr. Rajagovindan has stock in Vigil .
Simina Ticau Simina Ticau has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Simina Ticau has received stock or an ownership interest from Alnylan Pharmaceuticals.
Takanori Yokota, MD, PhD Dr. Yokota has nothing to disclose.
Alexandra Geffrey, MD Dr. Geffrey has received personal compensation for serving as an employee of Massachusetts General Hospital.
Andreas *Use 372386 Meier Andreas Meier has received personal compensation for serving as an employee of Voyager Therapeutics. Andreas Meier has received personal compensation for serving as an employee of Biogen.
Marie Ynez Davis, MD, PhD The institution of Dr. Davis has received research support from NIH NINDS. The institution of Dr. Davis has received research support from University of Washington Institute for Stem Cell and Regenerative Medicine. The institution of Dr. Davis has received research support from VA BLRD. Dr. Davis has received personal compensation in the range of $500-$4,999 for serving as a study section grant reviewer with NIH. Dr. Davis has received personal compensation in the range of $500-$4,999 for serving as a Grant reviewer with Parkinson's Foundation.
Sarah Wahlster, MD Dr. Wahlster has nothing to disclose.
Ali Toumadj, PharmD No disclosure on file
Janice W. Smith, PhD Dr. Smith has received personal compensation for serving as an employee of Roche Products Ltd. Dr. Smith has stock in Hoffman La Roche.
Andreas Meier, MD Dr. Meier has received personal compensation for serving as an employee of Vigil Neuroscience. Dr. Meier has received personal compensation for serving as an employee of Voyager Therapeutics. Dr. Meier has stock in Vigil Neuroscience. Dr. Meier has stock in Voyager Therapeutics.