Could a drug already available for cancer be used to treat some children with Noonan syndrome? This is what a retrospective ...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported fourth-quarter adjusted EPS of 92 cents, up from 49 cents reported a year ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

Total Revenues increased 16% year over year, driven by Voxzogo contributions from new patient starts in all ... for idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency ...

BioMarin Pharmaceutical Inc. ( NASDAQ: BMRN) Q4 2024 Earnings Conference Call February 19, 2025 4:30 PM ET Traci McCarty - Head of IR Alexander Hardy - President and CEO Brian Mueller - EVP and CFO ...

A diagnosis of NS in a patient with pulmonary stenosis or infant-onset hypertrophic cardiomyopathy would facilitate condition-specific counselling on outcome and prognosis. Noonan syndrome, an ...

"We begin 2025 ready to build on our leadership in treating genetically defined conditions and deliver breakthrough medicines to the patients we serve. This year, we expect a number of innovative ...

Pediatric endocrinologists are familiar with Noonan syndrome (NS) as the disorder represents one ... and a multidisciplinary approach is required to a proper patient management and care. In this ...

Immune cell populations of people with post-vaccination syndrome had lower levels of effector CD4 T cells and higher levels ...