Microbiota Transplant Therapy for Pitt Hopkins Syndrome - James Adams PhD, ASU @Synchrony2022

Pitt Hopkins Syndrome is a rare single-gene disorder that generally results in severe symptoms of intellectual disability, motor coordination impairments, autism, and chronic constipation.
Microbiota transplant therapy (MTT) involving vancomycin, bowel cleanse, and 12 weeks of microbiota capsules was used to treat 6 children with PTHS. The study design was Part 1: randomized, double-blind, placebo-controlled; Part 2: placebo group switched to real treatment; Part 3: long-term evaluation.
In part 1, group A (treatment) generally improved more than group B (placebo) on medication usage, GI symptoms, pain, average of 29 PTHS symptoms, and clinical global impressions of GI and PTHS symptoms.
In part 2 and 3, group A generally retained their improvements and sometimes improved more, at 3 and 6 months after treatment stopped.
In part 2, group B generally had additional improvements, and those improvements generally remained at end of part 3 (3 months after treatment stopped).
🎥 This talk was part of Synchrony 2022 Online Symposium - 'From Bench to Biopharma', organised by the The BRAIN Foundation
🎥 For more Synchrony 2022 talks and highlights:
• Synchrony Symposium 20...
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Synchrony synchronysympo... is the first and only international symposium on translational research in #autism, that brings together academia, #biotech, pharmaceutical companies and #venture partners from around the world with the mission to improve health and quality of life of people with #autismspectrumdisorder.
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The BRAIN Foundation brainfoundatio... is a 501c(3) non-profit. The founders of BRAIN envision a world where every child and adult on the autism spectrum is healthy, participates fully in education and employment, and has a better quality of life. It aims to catalyze research that results in evidence-based interventions for the disabilities associated with autism, and also results in better medical standard of care.
To accomplish this, it funds impactful research through #philanthropy and our network of partners in the venture, corporate, and grassroots community.