💉 Japan approved a $2 MILLION drug — covered by public insurance.
Elevidys, a gene therapy for Duchenne muscular dystrophy, is now the priciest medicine ever under Japan's universal healthcare.
One shot. One life changed.
#GeneTherapy #Elevidys #DuchenneMD #RareDiseases

Two Weeks After Ouster Prompted By Laura Loomer, Vinay Prasad Back At FDA - IndiaWest Journal News Two Weeks After Ouster Prompted By Laura Loomer, Vinay Prasad Back At FDA.

Two Weeks After Ouster Prompted By Laura Loomer, Vinay Prasad Back At FDA

Full Story: indiawest.com/two-weeks-af...

#VinayPrasad #FDAReinstatement #LauraLoomer #FDALeadership #VaccineRegulation #GeneTherapy #SareptaTherapeutics #DuchenneMD #FDAControversy

hey #DuchenneMD if #claymatthews is your dude? You are really hurting your charity-

Potential Treatment for Heart Disease Caused by Duchenne MD Catherine Jayasuriya thought it was strange her then-6-year-old son, Dusty Brandom, seemed to fall down more often than other children and couldn’t keep up with his siblings. That year, 1998, Jayasuri...

On #RareDiseaseDay, we honor the families who are fighting for a cure.
#DuchenneMD, a disease that weakens muscles & affects the heart. Research led by @eduardomarban.bsky.social offers hope through #CellTherapy slowing #HF. This breakthrough may extend lives & change futures.
tiny.cc/8hob001

Great talk by @eduardomarban.bsky.social at today’s @fda.gov workshop discussing his work on #CellTherapy for heart disease and the development of #CDCs used for treating #DuchenneMD.