XTalks

DECEMBER 18, 2024

CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. 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.

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

Pharmaceutical Technology

APRIL 24, 2023

In an email to this news service, João Conde, PhD, professor at NOVA Medical School, Universidade NOVA de Lisboa, said advances like the mRNA vaccines serve as a testament to the breakthroughs made by science over decades of research at the junction of genetics and nanomedicine. These could feature in the next four to five years.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

XTalks

SEPTEMBER 25, 2024

It is estimated that 900 people are living with NPC in the US, of which approximately one-third have been formally diagnosed with this ultra-rare, relentlessly progressive and fatal neurodegenerative disease. NPC is a rare genetic disorder that leads to progressive neurological symptoms and organ dysfunction.

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