When  risdiplam was approved by the FDA on August 7, 2020, the FDA announced it had also awarded a Rare Pediatric Disease Priority Review Voucher.  These vouchers are tradable, and have a value of roughly $100 million. The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation,…

Access for risiplam is limited in many countries. These are a few of the family fundraising pages. Poland.  https://www.gofundme.com/f/help-emilia-dembinska-sma-type-1 https://www.gofundme.com/f/hira-nur-sma-type2 India. https://milaap.org/fundraisers/support-keya-hatkar UK. https://www.gofundme.com/f/Support-Twins-Raffy-and-Siddys-SMA-1-Fight        

On November 18, 2022, Robert Sachs and Clare Love wrote to HHS Secretary Becerra, urging him to act on their patent to use US government rights in three patents on the prostate cancer drug Xtandi. Average Wholesale Price (AWP) price in the USA is $189,800 per year, 3 to 7 times the price is all…