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

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Dr. 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

Roots Analysis

AUGUST 12, 2024

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

Genetics 40

Genetics Gene Expression RNA DNA 40

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. It is characterized by a deficiency in molybdenum cofactor production.

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

It aids in optimizing individualized treatment plans by offering insights into brain volume to help inform the selection of the precise dose of the investigational product (IP). Importantly, FDA-approved products are now available for these PET imaging applications.

Trials 111

Trials Clinical Trials Clinical Trials Genetics 111

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. Oxlumo marks our third FDA approval in less than three years, positioning us to meet or exceed our Alnylam 2020 strategy and goals, and further highlighting the productivity of our RNAi platform and the speed at which we can bring innovative medicines to patients.

FDA Approval 52

FDA Approval Production RNA Medicine 52

Roots Analysis

AUGUST 31, 2023

Similarly, people suffering from lactose intolerance must avoid milk products. Quantitative real time polymerase chain reaction ( RT PCR ) and DNA microarray technologies have been successfully used to evaluate the interactions between genes and diet, which is measured in terms of changes in the genetic expression.

Genomics 40

Genomics Genome Genetics Gene 40

The Pharma Data

DECEMBER 9, 2020

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. It’s like taking out the trash.

Marketing 52

Marketing In-Vivo Protein In-Vitro 52

The Pharma Data

MARCH 16, 2022

As a manufacturer of custom oligonucleotides the company partnered in the development of molecular diagnostics and built a broad portfolio of diagnostic assays, in particular for inherited genetic as well as somatic mutation testing, quantitative assays for haematology and transplantation medicine.

In-Vitro 52

In-Vitro Genotype Reagent Protein 52

The Pharma Data

DECEMBER 9, 2020

The aim of the research will be to create a TGS IVD platform by optimizing a multi-layer approach encompassing the initial sample and library preparation, state-of-the-art sequencing technologies and improved genotyping procedures. To meet the increased demand, HALIX expands with two additional viral vector production, the company said.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52