Our Mission
The SSADH Association is a voluntary, not-for-profit, 501(c) (3) organization dedicated to helping children and families affected by an ultra-rare neurometabolic disorder known as Succinic Semialdehyde Dehydrogenase Deficiency or SSADH.
The SSADH Association is committed to those affected with this neurotransmitter disease through education, advocacy and research.
News & Updates
Galibra Neuroscience announces capsid license agreement for Gene Therapy
The SSADH Association funded the creation of a new mouse model to be created at Boston Children’s Hospital by Alexander…
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STTR Grant to Galibra and Boston Children’s Hospital
Galibra Neuroscience has been awarded $500,000 through the U.S. Department of Health and Human Services. This grant through the Small…
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Dr. Rotenberg is named 2025 Harrington Scholar-Innovator Award recipient.
Congratulations to Alexander Rotenberg, MD, PhD. for being named one of ten 2025 Harrington Scholar-Innovator Award recipient by the Harrington…
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Celebrating Rare Disease Day
Wisconsin mother creates SSADH Association after her son is diagnosed with neuro disorder. Spectrum News Wisconsin | BY Abbey Taylor |…
Read MoreOver a 100 patients, caregivers and doctors gathered from across the US, and from as far away as Asia, Europe and Greece to participate in the Externally Led - Patient Focused Drug Development (EL-PFDD) meeting. Additionally, we had participants join us virtually who were unable to travel because of the logistical challenges of traveling with their affected children.
The PFDD initiative was developed for the FDA to systematically obtain the patient perspective on specific diseases and their currently available treatments.
At the PFDD meeting the families highlighted the most significant symptoms of SSADH Deficiency and shared stories to reflect the impact that this disorder has on the patient’s daily life while emphasizing the lack of treatment options. The finding are summarized in
the Voice of the Patient report.
Video Recording From July 8, 2022 EL-PFDD Meeting
We are happy to provide the videos below of the
SSADH EL-PFDD Meeting, for those who were unable
to attend in person.
SSADH Association Welcome and Morning Session 1
EL-PFDD Meeting, Video 1 of 4.
Morning Session 2
EL-PFDD Meeting, Video 2 of 4.
Afternoon Session 1
EL-PFDD Meeting, Video 3 of 4.
Afternoon Session 2
