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Family Driven Development of Treatments for Rare Pediatric Neurological Diseases
Published article in the prestigious Clinical Pharmacology and Therapeutics Journal , titled “Family Driven Development of Treatments for Rare Pediatric Neurological Diseases https://onlinelibrary.wiley.com/share/author/CYASAK6VRRDKH23SJNZB?target=10.1002/cpt.2507
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The Year in Review summary 2021
The Year in Review summary 12/16/21. https://conta.cc/31S4v9J
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Sio Gene Therapies Announces Positive Interim Safety and Biomarker Data from Ongoing Phase
“Sio Gene Therapies Announces Positive Interim Safety and Biomarker Data from Ongoing Phase 1/2 Clinical Study of AXO-AAV-GM1 Gene Therapy in GM1 Gangliosidosis”. https://investors.siogtx.com/news-releases/news-release-details/sio-gene-therapies-announces-positive-interim-safety-and
Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease
Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis). Sio https://investors.siogtx.com/news-releases/news-release-details/sio-gene-therapies-announces-first-patient-dosed-clinical-trial?fbclid=IwAR2DaXAujFySSmtAoEIfDF1XhG3Gi56D6CgDkbMD2yWAFQ65kP28GV3G4B0
Taysha Gene Therapies “A Clinical Trial Option for Children with Tay-Sachs Disease or Sandhoff”
Taysha Gene Therapies “A Clinical Trial Option for Children with Tay-Sachs Disease or Sandhoff” https://www.tsd-sdgtxtrial.com/?fbclid=IwAR39L0MTuN_B-yXzwFtR-6ifmNtl_evd_CFqduSRfqe4enJckIsCVfG3pFk
Axovant Gene Therapies Announces FDA Clearance of IND for AXO-AAV-GM2 Gene Therapy in Tay-Sachs and Sandhoff Diseases
AXO-AAV-GM2 is the first investigational gene therapy to receive IND clearance in Tay-Sachs and Sandhoff diseases Represents Axovant’s second IND clearance for a gene therapy program in the last 12 months NEW YORK, Nov. 09, 2020 (GLOBE NEWSWIRE) — Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the U.S. Food
TEAM DAD Parenting Series: Dad Highlights Program
TEAM DAD Parenting Series: Dad Highlights Program GAME ON FOR FATHERS OF CHILDREN WITH SPECIAL NEEDS! Join us on November 5th at 7:00pm as we have an in-depth conversation with a father who has taken his experiences as a parent of a child with special needs and created change in his community and in the
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A Beacon of Hope: Barry Byrne, M.D., Ph.D., receives “See The Light” Award from the Mathew Forbes Romer Foundation
Barry Byrne, M.D., Ph.D., receives “See The Light” Award from the Mathew Forbes Romer Foundation By: Tyler Francischine Oct. 15, 2020 — For families facing a devastating diagnosis of pediatric neurodegenerative genetic disease, Barry Byrne, M.D., Ph.D., provides a sense of hope and possibility. As director of the Powell Gene Therapy Center, the associate chair
The Mathew Forbes Romer Foundation Inc Receives 2020 Best of Boca Raton Award
Boca Raton Award Program Honors the Achievement BOCA RATON February 10, 2020 — The Mathew Forbes Romer Foundation Inc has been selected for the 2020 Best of Boca Raton Award in the Education & Research Foundation category by the Boca Raton Award Program. Each year, the Boca Raton Award Program identifies companies that we believe
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