XTalks

DECEMBER 18, 2024

It also received the FDAs rare pediatric disease priority review voucher along with the approval, which will help expedite the agencys assessment of another application. Neurocrines share price increased by six percent after news of the approval.

Genetics 116

Genetics Hormones Trials Drugs 116

Pharmaceutical Technology

APRIL 24, 2023

Covid-19 vaccines from Moderna and Pfizer /BioNTech, which use lipid nanoparticles, became the only two FDA-approved vaccines for almost all ages. However, after a period of slow movement in the field, the FDA approved the first nanodrug, Doxil, for Kaposi sarcoma in 1995.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

XTalks

SEPTEMBER 25, 2024

While Miplyffa will be the only treatment for NPC with neurological manifestations on the market, Sanofi has an infusion called Xenpozyme for non-central nervous system manifestations of Niemann-Pick, also known as acid sphingomyelinase deficiency (ASMD), which got an FDA green light in 2022.

Genetics 52

Genetics Drugs Trials Pharmacy 52

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95