Company Exec. Chairman & CEO in a fireside chat is expected to highlight the role of Skinject in an autosomal dominant rare disease, AI-enabled drug development strategy and partnering readiness ...

Atrium Therapeutics, Inc. (Nasdaq: RNA) launched today as a newly independent, publicly traded company dedicated to delivering RNA therapeutics directly to the heart to transform care for people ...

BioMarin’s Voxzogo (vosoritide) won FDA approval in paediatric achondroplasia in November 2021 and secured a label extension ...

Atrium Therapeutics launched today as a newly independent, publicly traded company dedicated to delivering RNA therapeutics directly to the heart to transform care for people living with rare, ...

Atrium Therapeutics Inc. has launched as a newly independent, publicly traded company dedicated to delivering RNA therapeutics.

"Monogenic" diseases, triggered by mutations in just one gene, may actually be more complex than scientists thought.

Learn more about whether Crinetics Pharmaceuticals, Inc. or Prestige Consumer Healthcare Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial ...

Franzen wants to move DuPont from the materials sector, where it is considered a specialty chemicals company, to somewhere in ...

Patients with macular inherited retinal disease are significantly less likely to receive definitive genetic testing results than peripheral IRD patients.

More than 30 rare muscular dystrophy types cause progressive muscle weakness from inherited gene mutations. Combined, they affect about 1 in 5,000 to 8,000 people. Common subtypes vary by age of onset ...

Retex Pharmaceuticals Inc. has patented vasopressin V2 receptor antagonists potentially useful for the treatment of hyponatremia, cardiovascular disorders, autosomal dominant and autosomal recessive ...

GRN patients in Cohort 3 of ongoing upliFT-D study Treated first FTD-C9orf72 patient with Dose 2 PBFT02 in Cohort 4 of upliFT-D study On track to report updated interim safety and biomarker data from ...