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 ...

MUSCAT: A collaborative arrangement reached between National Pharmaceutical Industries Company (NPI) – the first ...