GM1 Gangliosidosis Disease Overview - Dr. Cynthia Tifft

The GM1 Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD) was held on Friday, October 14th as part of the Cure GM1 Foundation 2022 virtual community conference. Dr. Cynthia Tifft provided a disease overview of GM1 Gangliosidosis.
Please always consult your physicians regarding medical decisions and clinical trials. The Cure GM1 Foundation does not prescribe medications or treatments and we can not specifically endorse a treatment which is not FDA-approved. This information is being shared for informational purposes for you to discuss with your doctors.
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[@shakildanish0418]

My child has gm1 disorder can any body help.

[@curegm1foundation]

So sorry to hear that. We have a private Facebook group for families. Right now, none of the clinical trials are enrolling. Passage Bio plans to reopen their trial in the end of 2023.