approves-first-treatment-inherited-

rare-disease
o https://www.fda.gov/industry/
PORPHYRIA LINK: medical-products-rare-diseases-and-
conditions/designating-orphan-
YOUTUBE product-drugs-and-biological-
products
o https://www.youtube.com/watch? o
v=1vIdP0SSC4g
o https://www.youtube.com/watch?
v=HOJhGFMHjsQ
o https://www.youtube.com/watch?
v=gJpUyP6oFAA
o https://www.youtube.com/watch?
v=FxAYQgLmv8o
o https://www.youtube.com/watch?
v=yuLncJa_t94

RESEARCH:

o https://www.mayoclinic.org/
diseases-conditions/porphyria/
symptoms-causes/syc-20356066
o https://www.mayoclinic.org/
diseases-conditions/porphyria/
symptoms-causes/syc-20356066
o https://
accessmedicine.mhmedical.com/
Content.aspx?
bookId=392&sectionId=41138853#5
6066570
o https://
www.genomicseducation.hee.nhs.uk
/blog/nice-approves-rnai-therapy-
for-porphyria/
o https://www.dovepress.com/
spotlight-on-givosiran-as-a-
treatment-option-for-adults-with-
acute-hep-peer-reviewed-fulltext-
article-DDDT
o https://www.fda.gov/news-events/
press-announcements/fda-