The Give Kids a Chance Act would create incentives for pharmaceutical companies to develop treatments for children and speed up access to treatments that have been improved in adults but haven’t been tested in kids.

Orphan Drug Act that made porphyria treatments possible. Some members of Congress want to strip the Orphan Cares Act out of the law by passing a repeal.

Right now, proposed budget cuts threaten to slash NIH funding by as much as 40%. For rare diseases like porphyria, this is devastating. Here’s how you can help.