XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

JULY 3, 2023

PrecisionLife and the University of Oxford have signed a data access agreement to license the Oxford Endometriosis Gene (OXEGENE) dataset to develop new personalised treatments for endometriosis patients. Endometriosis affects 10% of women globally, but on average it takes over seven years for patients to receive a diagnosis.

Genetics 52

Genetics Genotype Medicine Licensing 52

The Pharma Data

NOVEMBER 2, 2020

ONWARD is investigating Adial’s lead drug candidate, AD04, as a therapeutic agent for the treatment of Alcohol Use Disorder (AUD) in persons with certain target genotypes related to the serotonin transporter and receptor genes. Key ONWARD highlights include: All 25 planned investigative sites are active.

Trials 52

Trials Genotype Marketing Production 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

XTalks

APRIL 15, 2021

A 2015 study published in Nature Genetics found that the availability of human genetic data made investigational drugs twice as likely to pass pivotal trials and eventually be approved. Figure 1: The use of Mendelian randomization to validate genetic drug targets.

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.

Genome 98

Genome Genomics Medicine Genetic Disease 98

Roots Analysis

AUGUST 31, 2023

According to a study, around 20,000 genes are present in the human body, all of which interact with the nutrients in the food, either directly or indirectly. For instance, carbohydrates increase the expression of genes responsible for the synthesis of enzymes and transporters involved in their metabolism.

Genome 40

Genome Genomics Genetics Gene 40