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

SEPTEMBER 15, 2020

A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. Specifically, the vaccine contains human recombinant glutamate decarboxylase 65 (GAD65) protein conjugated to aluminum hydroxide (GAD-alum).

Vaccination 103

Vaccination Vaccine Insulin Trials 103

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. One substantial obstacle is from the additional regulatory requirements of gene therapies containing genetically modified organisms (GMOs). Rare diseases can often be progressive, chronic and fatal.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

FEBRUARY 4, 2021

Through these collaborations, cohort research dataset sharing programs allow for whole-genome sequence and genotype data to be made available for independent research. Now, if all you’ve already got is a bioresource of 20,000 people that have already been genotyped, you can choose those from that sample and focus your effort.”.

Research 98

Research Biobanking Drugs Genome 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

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

Drug Discovery World

JULY 15, 2022

The new sequences contain largely highly repetitive DNA structures such as centromeres, telomeres, acrocentric genomic regions, where massive arrays of tandem repeats predominate, and heterochromatin. iv) Highly repetitive regions contain some of the highest mutation rates.

Genome 52

Genome Genomics DNA Containment 52