News on rare diseases, including the development ... The page features in-depth reporting on genetic diseases, immunodeficiencies, metabolic disorders and more.
Get Instant Summarized Text (Gist) A new approach using personalized antisense oligonucleotides (ASOs) and patient-derived organoids offers promising treatment options for rare genetic diseases ...
Rare diseases are typically due to genetic causes. These causes are more and more often identified using genome sequencing techniques. Genome sequencing examines the entire genome of an individual ...
SINGAPORE – Inherited retinal diseases (IRD) are not as rare here as previously thought, a research programme at the Singapore National Eye Centre (SNEC) has found. IRDs are a group of genetic ...