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. How Can Study Protocols Be More Effective?

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. The FDA remains committed to facilitating the development and approval of safe and effective therapies for patients affected by rare diseases — an area of critical need.”. The approval was granted to BridgeBio Pharma, Inc.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

APRIL 15, 2021

million people in the US, yet there is no treatment for the most common form of the disease: heart failure with preserved ejection fraction (HFpEF). A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl. Heart failure affects about 6.2

Development 98

Development Drugs Genome Genomics 98

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Oxlumo should be administered by a healthcare professional.

FDA Approval 52

FDA Approval Production RNA Medicine 52