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.

A clinical trial is one that involves human participants and seeks to answer specific questions about a type of medical intervention. This can be a drug or other type of treatment, such as nutritional ...

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 ...

Pneumonia symptoms can vary based on the cause and severity of the lung infection. For some, it might feel similar to the flu or be barely noticeable. For others, pneumonia can cause severe ...

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 ...

announced the initiation of a clinical trial for YOLT-204, an investigational therapy for the treatment of transfusion-dependent beta-thalassemia (TDT). TDT is a severe genetic blood disorder ...