NINDS Awards two year grant: ERT for SSADH Deficiency

January 11, 2022

Granted awarded for Developing an inducible mouse model for gene replacement therapy in Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD) Drs. Henry Lee and Alex Rotenberg of Boston Children’s Hospital were awarded $221,250 for a two year period ending 12/31/2023 from the National Institutes of Neurological Disorders and Stroke (NINDS).  Below is the…

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Proceedings Published from Virtual Conference

December 14, 2021

The manuscripts from the 2020 Virtual SSADH Conference are being published on-line through the Journal of Child Neurology.  The majority of the topics are from the presentations, however when we moved from an in-person to a virtual event we were forced to scale back and eliminate some of the presentations. …

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Wave of Hope

December 11, 2021

The Le Vine family held a Wave of Hope – SSADH Fall Harvest Beach fundraiser on Sunday, October 17 from 1 – 5 pm at the Columns Restaurant in Avon overlooking the ocean. The popular band Spare Parts is played and there was a gift auction as well. The even…

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Conference on Rare Neurotransmitter Diseases

December 4, 2021

Announcement and Invitation on behalf of Lil’ Brave One:   Rare Neurotransmitter Diseases: from Novel Research to Focused Treatments through iNTD Network Strengthening and Patient Empowerment Save the date September 29th 2022 – October 1st 2022 We are pleased to announce that the conference on rare neurotransmitter diseases will take…

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GC Pharma and Speragen Announce Joint Development of SSADHD Enzyme Replacement Therapy and License Agreement of the patent of SSADH Protein generation plasmid

July 20, 2021

GC Pharma and Speragen Announce Joint Development of SSADHD EnzymeReplacement Therapy and License Agreement of the patent of SSADH Proteingeneration plasmid Collaboration to focus on developing First-in-Class drug to fulfill unmet needs of patients YONGIN, SOUTH KOREA & AUSTIN, TX, USA, 19 July 2021 – GC Pharma (006280.KS)and Speragen, Inc.…

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The SSADH Association awarded $55,000 to Henry Lee, Ph.D

July 6, 2021

The SSADH Association awarded $55,000 to Henry Lee, Ph.D., Alexander Rotenberg, Ph.D., MD, and Phil Pearl, MD from Boston Children’s Hospital / Harvard Medical School to continue evaluating the potential for Enzyme Replacement Therapy for SSADH patients. The grant period will begin July 1st and run for six months to evaluate the optimal…

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