Rare & Genetic Diseases

A group of researchers leveraged their location to tackle questions about the rare autoimmune disorder, Addison’s disease.

Different environmental exposures, different lifestyles, and so on. So they might have different weights or different styles or even different chronic diseases.

“Gene therapy has huge potential. It has already led to cures for conditions such as spinal muscular atrophy (SMA). In the future, we will be looking at conditions like motor neurone disease, Alzheimer’s and rare cancers,”

According to the company, the new data underscore the critical importance of identifying and treating SMA as early as possible.

Advances in genomics research increased the speed with which vaccines came to the fore to mitigate infection by the SARS-CoV-2 virus, thanks to early sequencing of the virus. Here is a quick roundup of some recent genomics news related to COVID-19.

Genomics, proteomics, and transcriptomics loom large in oncology

New AI tool to be tested in NIH-funded study to improve diagnosis of Parkinson’s and related disorders

LIVE April 30th at 2:30 PM ET.
In this webinar, researchers will discuss how gene therapy has been used to treat disease and new research approaches to regenerative gene therapy.

EMA grants Orphan Drug designation to JR-171 for the treatment of mucopolysaccharidosis type I