Promising Pathway Act

The Foundation is very pleased to be supporting this very important act. Why is this relevant to H-ABC/Tubb4A? Clinical trials undergo multiple phases, the first of which is safety, then evidence that specified endpoints or biomarkers are met. Trials often stall due to the difficulty in meeting these FDA requirements. This Act will allow access to a drug if it was proven as safe, but didnt get to commercialization due to the rigid FDA requirements.

https://www.youtube.com/watch?v=HgcRCKdf9qg

One Pager on the bill - https://www.braun.senate.gov/wp-content/uploads/2024/05/PPA-2.0-One-Pager.pdf

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How Microtubules work and what causes the disruption in the Tubb4a gene causing H-abc/Tubb4A related leukodystrophy

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Peace of Woodstock Fundraiser