Despite promising Phase 3 results, safety concerns, namely liver injury, have impacted Agios stock and could affect Pyrukund's approval and market adoption. Read the full report here.

ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused –– ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent beta-thalassemia ... exceptional efficacy in clinical trials.

Ocular manifestations of SLE are common and may lead to permanent blindness from the ... The terminology of ‘vasculitis’ in lupus retinopathy can be confounding among clinical presentation and ...

Metabolic syndrome affects between 30% and 40% of adults by age 65. It's rare to have metabolic syndrome symptoms—so the diagnosis depends on blood tests and other clinical measures. Lifestyle changes ...

Concerns about treatment were associated with lower quality of life and more symptoms of anxiety and depression. Conclusions Despite their requirement for multimodal therapy, thalassemia patients ...

The Alpha Thalassemia market is expected to experience substantial growth, driven by the rising demand for effective therapies, advances in genetic research, and ongoing clinical trials focused on ...

Walilko knew something was wrong—yet says clinicians continued to brush off her symptoms. After doing internet ... Sinai Medical Center and senior clinical researcher at the National Heart ...

The Alpha Thalassemia market is expected to experience substantial growth, driven by the rising demand for effective therapies, advances in genetic research, and ongoing clinical trials focused on ...