The phase 3 STEER study enrolled 127 treatment-naïve patients aged 2 to less than 18 years with SMA type 2 who were able to sit but had never walked independently.

The researchers found that between 1999 and 2022, there were 63,550,429 deaths in the United States, of which 0.06% had cold recorded as an underlying or contributing cause of death. Cold-related ...

Health sector's share of the economy was similar in 2023 and 2022, but lower than in 2020 and 2021, during height of COVID-19 ...

The approval was based on data from the randomized, placebo-controlled, double-blind, phase 3 RATIONALE-305 trial.

The approval was based on data from the randomized, double-blind, placebo-controlled phase 3 URO-901-3005 trial.

According to Vertex Pharmaceuticals, approximately 300 additional people with CF are now eligible to receive treatment.

The resubmitted NDA for sotagliflozin for adults with T1D and CKD included post hoc analyses from the TANDEM clinical development program and post hoc analysis from the SCORED trial.

The ongoing shortage of liraglutide injection and other GLP-1 medications prompted the FDA to prioritize the generic drug applications for these medications in order to improve patient access.

(HealthDay News) — For adults with migraine, atogepant 60mg once daily was superior to placebo for efficacy and functional measures of treatment across 3 trials, according to research published online ...

Results from the VENTURE trial showed significant reductions in weight and hunger among younger patients treated with ...

The BLA acceptance was supported by data from the randomized, placebo-controlled phase 2b/3 CLEVER trial as well as interim ...