pharmaphorum

JANUARY 18, 2023

Pharmaceutical companies and biotechs are also adapting their approaches, launching patient finding and engagement programmes that can start years before clinical trials begin and allow them to run ‘recontact by genotype’ studies that the Resilience Project would have liked to do. Giving participants something in return.

Genetic Disease 80

Genetic Disease Genetics Research Genome 80

XTalks

APRIL 15, 2021

Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. The reverse could also be true whereby the development of coronary artery disease may impact heart rate. HFrEF vs. HFpEF.

Development 98

Development Drugs Genome Genomics 98

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases. ” or “Why is my child different?”

Genome 98

Genome Genomics Medicine Genetic Disease 98

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

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

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. Together, the ILLUMINATE studies comprise a comprehensive clinical development program intended to demonstrate the safety and efficacy of Oxlumo across the full spectrum of patients diagnosed with PH1.

FDA Approval 52

FDA Approval Production RNA Medicine 52