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. The company said Crenessity will be available through specialty pharmacy PANTHERx Rare in order to centralize and simplify fulfillment of prescriptions for the drug.

Genetics 116

Genetics Hormones Trials Drugs 116

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases. Through the collaboration, the companies focus on connecting clinical trial data with specialty pharmacy information, natural history, registry data, and broad claims databases.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

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

XTalks

SEPTEMBER 25, 2024

Zevra said it expects Miplyffa to be available in US pharmacies in the next eight to 12 weeks and is gearing up for launch activities immediately. If you want your company to be featured on Xtalks.com, please email ayeshar@xtalks.com.

Genetics 52

Genetics Drugs Trials Pharmacy 52

Pharmaceutical Technology

APRIL 24, 2023

If successful, such applications could be used for neurodegenerative conditions like Alzheimer’s disease. Libera Bio is using these nanocarriers , developed by the company’s cofounder MJ Alonso, PhD, professor, Department for Pharmacy and Pharmaceutical Technology, Universidade de Santiago de Compostela, to deliver antibodies.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147