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 97

Genetic Disease Genetics Research Genomics 97

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. HS: Absolutely.

Genome 98

Genome Genomics Medicine Genetic Disease 98

XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Hylton V. The approval was granted to BridgeBio Pharma, Inc. Nasdaq: BBIO ) and its affiliate Origin Biosciences, Inc.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

Roots Analysis

AUGUST 12, 2024

Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine. Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities.

Genetics 40

Genetics Gene Expression RNA DNA 40

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. On November 19, the European Commission granted marketing authorization for Oxlumo for the treatment of PH1 in all age groups, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). President of R&D at Alnylam.

FDA Approval 52

FDA Approval Production RNA Medicine 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