A retrospective cohort study shows that renal transplantation appears to be safe and effective in the treatment of renal disease from cystinosis.
The price for treating patients with homozygous familial hypercholesterolemia (HoFH) may dramatically drop based on the approval of Repatha (evolocumab) injection by the FDA.
Matthew Sherman, MD, chief medical officer at Acceleron provides an overview of those products (sotatercept, dalantercept, and ACE-083) in addition to their lead product luspatercept.
Phase 3b/4 data published in the New England Journal of Medicine, shows that combining two approved orphan drugs for pulmonary arterial hypertension (PAH)— ambrisentan with tadalafil—is significantly superior to each drug alone.
Health officials have declared that a recent outbreak of Legionnaire’s disease in the South Bronx is officially over. The outbreak began in the South Bronx this July, sickening more than 120 and killing 12
Barry Greene of Alnylam Pharmaceuticals explains the various forms of transthyretin amyloidosis and the RNAi drugs in development to treat these conditions.
Chris Garabedian talks about the numerous orphan drugs in development for Duchenne muscular dystrophy (and yes, he does have a favorite among them).