This expanded indication enables children with this devastating diagnosis to be treated as early as possible, when there is the greatest potential to impact the natural course of disease.
Tag Archive for: pediatrics
The company said an antibody-based drug met the main goal of a mid-to-late stage trial testing it as a therapy to protect infants from disease caused by the respiratory syncytial virus.
In a political climate where discourse is often marked by division, there remain crucial issues that bridge partisan divides. The vital importance of treating children living with rare diseases is certainly one such cause.
The U.S. health regulator has declined to approve Rocket Pharmaceuticals’ gene therapy to treat a rare and severe pediatric disorder that causes the immune system to malfunction.
Yesterday Tris Pharma secured the FDA’s green light for Onyda XR, the first liquid non-stimulant nighttime treatment for attention deficit hyperactivity disorder in pediatric patients.
The FDA on Tuesday approved Day One Biopharmaceuticals’ type II RAF inhibitor Ojemda, which is designed to penetrate the blood-brain barrier, for the treatment of relapsed or refractory pediatric low-grade glioma.
The World Health Organization is likely to issue a wider warning about contaminated Johnson and Johnson-made children’s cough syrup found in Nigeria last week, it said in an email.
The batch recalled was made by J&J in South Africa in May 2021 but Kenvue now owns the Benylin Paediatric syrup brand after a spin-off from J&J last year.
Gilead Sciences’ Vemlidy on Thursday secured a label expansion from the FDA, allowing the antiviral drug’s use to treat chronic hepatitis B in pediatric patients aged six years and older.
Orchard Therapeutics, a unit of Japan’s Kyowa Kirin , priced its newly approved gene therapy for a rare, life-threatening genetic disease on Wednesday at $4.25 million, making it the most expensive drug in the United States.