Nobias Therapeutics, a clinical-stage company developing novel small-molecule therapeutics for rare diseases, today announced that it has secured preliminary alignment with the U.S. Food and Drug ...

Researchers have identified a promising strategy to improve memory and brain cell function in models of 22q11.2 Deletion Syndrome. The study, published in eLife, shows that reducing the levels of ...

ABSTRACT: Background: DiGeorge syndrome (also known as velo-cardio-facial syndrome) is a rare multisystem genetic disorder occurring in approximately 1 in 4000 to 1 in 6000 live births [1]. Although ...

A recent study led by UC Davis Health researchers provides new insights into the molecular changes linked to the rare genetic condition 22q11.2 deletion syndrome, or 22q. It found unique ...

About 22q11.2 Deletion Syndrome 22q11.2 deletion syndrome (22q) is a disorder caused by a small missing piece of the 22nd chromosome. The deletion occurs near the middle of the chromosome at a ...

("Harmony" or the "Company") (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, will present ...