Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia 0 genotypes. thalassemia in adult and paediatric patients.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

XTalks

JANUARY 21, 2025

TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Date of FDA approval : March 29, 2024 Approved for : To detect exposure to human parvovirus B19. TriClip size comparison photo. Photo courtesy of Abbott Medical.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

FDA Approval 52

FDA Approval Production RNA Medicine 52

XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

Roots Analysis

APRIL 28, 2024

Factors Contributing to Kidney Diseases There are several factors which contribute to an increased risk of developing kidney diseases, including genetic causes and underlying metabolic causes ( diabetes, high blood pressure, aging and obesity ), which when combined with other factors increase the risk of developing kidney diseases.

Genetics 40

Genetics Genotype Development Research 40

XTalks

DECEMBER 12, 2023

Importantly, FDA-approved products are now available for these PET imaging applications. The approvals have unlocked the potential to use these highly sensitive agents to assess patient eligibility, determine target engagement and evaluate treatment efficacy for Alzheimer’s disease.

Trials 111

Trials Clinical Trials Clinical Trials Genetics 111

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95