News

One Million Dollars awarded to SSADHD Gene Therapy

June 12, 2024

Drs. Lee & Rotenberg were awarded approximately one million dollars for the next three years to fund their continued work on Gene Therapy. The funding will be coming through an…

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Calling for Reform in US Newborn Screening

May 3, 2024
Newborn Screening

Alice McConnell presented at the World Orphan Drug Congress in Boston on April 25th.  We are grateful to Alice who took the opportunity to speak out about the need to…

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Gene Replacement Therapy Update

May 3, 2024

Drs. Henry H. C. Lee, Itay Tokatly Latzer, Mariarita Bertoldi, Guangping Gao, Phillip L. Pearl, Mustafa Sahin, and Alexander Rotenberg teamed up to create a paper on the status of their…

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GC Biopharma builds mRNA manufacturing plant in South Korea

January 12, 2024

The plant features a single-use manufacturing unit, reducing the risk of cross-contamination and facilitating the production of a number of products simultaneously. GC Biopharma builds mRNA manufacturing plant in South…

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SSADH Presented at AES

January 5, 2024

The American Epilepsy Society (AES) annual meeting for 2023 was held in Orlando, Florida from December 1-5, 2023.  The event took place at the Orange County Convention Center bringing together…

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Neurobiology of SSADH Deficiency

January 5, 2024

Over the past three and half years, Wardiya Afshar-Saber, Ph.D. a research fellow in Neurology at Boston Children’s Hospital in the Sahin Lab has been focusing on establishing a platform…

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SSADH Deficiency becomes the topic for the Boston Children’s Hospital Metabolism Grand Rounds.

November 29, 2023

On Tuesday November 28th Dr. Phillip L. Pearl gave a talk, titled: Results of the SSADH Deficiency Natural History Study. This talk included a 1)     Review of the clinical, imaging, and neurophysiologic outcomes…

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Wave of Hope Fundraiser

September 22, 2023

Amber and Haley, are two young, inspiring women who have an extremely rare metabolic disease called Succinic Semialdehyde Dehydrogenase Deficiency (SSADH); there are only 400 cases of SSADH worldwide. Amber…

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Voice of the Patient Report Submitted to FDA

May 5, 2023

We are happy that after hosting the Externally Led Patient-Focused Drug Development (EL-PFDD) meeting, in July of 2022, the Voice of the Patient report has been submitted to the FDA’s…

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Conquering a rare metabolic condition: A family, a pediatrician, and two labs join forces

May 2, 2023

Boston Children’s Hospital and Dr. Phil Pearl, have long been at the center or our mission to find a treatment for SSADH Deficiency. Pearl teamed up with Mike Gibson, PhD.…

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