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
The Year in Review summary 12/16/21. https://conta.cc/31S4v9J
“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 (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” https://www.tsd-sdgtxtrial.com/?fbclid=IwAR39L0MTuN_B-yXzwFtR-6ifmNtl_evd_CFqduSRfqe4enJckIsCVfG3pFk
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 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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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 …
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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South Florida-Based Organization Hosts Annual “Sweetness and Laughter” Gala & “See the Light” Awards Ceremony March 15th at the Broward Center for the Performing Arts Boca Raton, FL, Jan. 17, 2020 – Twenty-two years after The Mathew Forbes Romer Foundation, or MFRF, was founded, the South Florida nonprofit organization dedicated to education and research into …
