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

Roots Analysis

AUGUST 12, 2024

Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities. Protein Based Assays- These tests measure the presence and levels of specific proteins, which can serve as biomarkers for various diseases. Examples includes microarrays and fluorescence in situ hybridization.

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. About Primary Hyperoxaluria Type 1 (PH1) PH1 is an ultra-rare genetic disease that affects an estimated one to three individuals per million in the United States and Europe. Oxlumo should be administered by a healthcare professional.

FDA Approval 52

FDA Approval Production RNA Medicine 52