The fall in AGIO stock comes after reports of an unexpected side effect linked to its sole-marketed drug Pyrukynd in thalassemia patients.

Sometimes, people with iron deficiency anemia don't respond to treatment with iron supplementation and may go months to years with little to no improvement in anemia. This can happen if the dose is ...

ENERGIZE-T Study Achieved Primary and All Key Secondary Endpoints in Adult Patients with Transfusion-Dependent Alpha- or Beta-Thalassemia –– ...

A B12 deficiency in females can cause symptoms such as fatigue, tingling feet, or depression. The symptoms of a B12 deficiency are not necessarily any different between males or females.

Lindy Noel's mom always knew her daughter was going to change the world. She just didn't know it would be the world of NHL ...

So, too, has Porty learned to manage his chronic case of pyruvate kinase deficiency (PKD), not to be confused with polycystic kidney disorder (also PKD, which we'll get to in a minute).

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable ...

Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD ...

A vitamin B12 deficiency can lead to nerve and brain damage and anemia. Early symptoms include weakness, constipation, and depression. It can result from dietary factors, medication use ...

Despite a delay in the RP-L301’s pivotal study for Pyruvate Kinase Deficiency (PKD), these developments collectively reduce the risks associated with the ongoing pivotal DD study, justifying the ...

Pyruvate kinase deficiency (PKD) is an autosomal recessive genetic disease caused by mutations in the PKLR gene. To date, the clinical manifestations of PKD are heterogeneous, ranging from fetal ...