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 ...

Drug coverage for four drugs used to treat some rare ... genetic conditions. Individual provinces have been signing agreements with the federal government under a $1.4 billion rare disease ...

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...