Astellas’s update on the Fortis clinical trial of AT845 in adults for pompe disease
The FDA informed Astellas that it did not have sufficient information to assess the risks to subjects
The FDA informed Astellas that it did not have sufficient information to assess the risks to subjects
If approved, Tafinlar + Mekinist may offer a potential new standard-of-care for pediatric patients with this brain cancer
GSK to pay $2.1 billion upfront and up to $1.2 billion in potential development milestones
Avillion announces expansion of partnership with AstraZeneca on PT027 and will fund and execute a new Phase 3b study
68% of patients receiving Kymriah in the ELARA trial experienced a complete response, with an 86% overall response rate, along with a remarkable safety profile
PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas administered by intravenous injection
Three RFPs now open for qualified researchers through NORD's Jayne Holtzer rare disease research grants program
A Promising Innovative Medicine (PIM) designation is an early indication that leniolisib is a candidate for the MHRA's Early Access to Medicines Scheme
This product is indicated for the short-term treatment of a breathing problem (apnea) in premature infants
Ondexxya is the first approved reversal agent specifically for Factor Xa inhibitors, providing a major advance in the treatment of patients hospitalised with life-threatening bleeding
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