May Newsletter

Are you a bike rider, scooter driver, stroller pusher, couch napper, or pet promenader? You can use any of those everyday skills to help defeat APBD! Come join the APBD “Tour de Friends” riding team in the Million Dollar Bike Ride on June 12 and help raise needed funds for rare disease research!
You don’t need to ride with wheels – although you certainly can do that. You do not need to raise funds yourself – but you can do that, too! By joining APBD Tour de Friends, you can help ensure our success. As captain of this team, I’m recruiting YOU!

Over the past five years, 172 APBD Tour de Friends riders have raised more than $540,000 to fund nine APBD research grants, supporting the development of compounds and gene therapies – and moving critical APBD research closer to clinical trials.

Join us in our vision of a world without APBD - let’s roll!
Harriet Saxe
Captain, APBD Tour de Friends
APBD Research Foundation Board Member
(Editor’s Note: As APBD gains wider attention among the public at large, we want to share the latest developments in APBD research, key events and partnerships, and other information. APBD in the News… is our new feature which we hope you’ll take the time to read and, most importantly, share! – Jeff)
APBD Featured in the Association for Glycogen Storage Disease’s Newsletter

Last month, the Association for Glycogen Storage Disease – one of our partnering organizations – featured an APBD article in their newsletter, The Ray. The story gives an overview of the various clinical presentations of Glycogen Storage Disease IV (GSD IV) – including APBD – which are caused by autosomal recessive mutations in the glycogen branching enzyme (GBE1) gene. It is based on a scientific article – published in the Journal of Inherited Metabolic Disease – by Dr. Paulo Victor Sgobbi Souza at the Federal University of São Paulo in Brazil. Read more…
Taysha Gene Therapies Announces New Data on Multiple Preclinical Programs and Upcoming R&D Day
Taysha Gene Therapies, Inc., recently announced new data for multiple preclinical programs – including TSHA-112 (an intrathecally delivered AAV9 viral vector designed for miRNA-mediated knockdown of the GYS1 gene) to treat APBD. Taysha is a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic central nervous system (CNS) diseases in both rare and large patient populations. In June, Taysha will host a virtual R&D Day, where they will highlight progress across their research and development pipelineRead more…

From Chat events to workshops and scientific symposiums, the Foundation hosts programs for patients, caregivers, and health professionals in an effort to build community and collaboration. Our upcoming events include:
Patient Chat
June 23, 2021, 8pm ET/5pm PT
Caregiver/ Family Chat
June 10, 2021, 8:30pm ET/5:30pm PT
To learn more about these events and to participate, email us at
ImmunoBrain Checkpoint

This month’s spotlight is on ImmunoBrain Checkpoint (IBC), a Platinum Parsnip sponsor of our recent ROOTS event.
Founded in 2015, IBC is an Israeli biopharmaceutical company that develops novel neuroprotective immune therapies for neurodegenerative diseases. The company’s therapies under investigation boost the immune system, helping to protect and repair the brain and the central nervous system. IBC developed a proprietary fully human anti-PD-L1 antibody, engineered based on its mechanism of action to treat Alzheimer’s disease and dementia. IBC plans to start its first-in-human clinical trial in 2021.
“Age-related immune dysfunction is a major contributor to the onset and progression of neurodegenerative disorders,” explained Nathan Hevrony, co-founder and executive chairman of IBC.
IBC's novel approach for the treatment of neurodegeneration is based on years of innovative, cutting-edge scientific discoveries made in the lab of Michal Schwartz, Professor of Neuroimmunology at the Weizmann Institute of Science. More information about IBC can be found at
Thank you to all of our ROOTS sponsors!