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

(Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering ... in children aged 1 to <18 years with PK deficiency who are not regularly transfused achieved ...

USA-based Agios Pharmaceuticals has announced that the ACTIVATE-Kids Phase III study of mitapivat in children aged 1 to <18 ...

Q4 2024 Earnings Call Transcript February 13, 2025 Operator: Good morning, and welcome to Agios’ Fourth Quarter 2024 ...

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) announced that the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to ...

Presented Phase 1 clinical results on tebapivat (AG-946) in patients with sickle cell disease at ASH 2024. Pediatric Pyruvate Kinase (PK) Deficiency: Reported in a separate press release today, ...

Agios Pharmaceuticals公司日前宣布，“first-in-class”口服丙酮酸激酶（PK）激活剂mitapivat，在3期临床试验ACTIVATE-Kids中达到主要终点。这项研究评估了mitapivat治疗1至18岁PK缺乏症儿童的效果，这些患者不经常接受输血。试验数据显示，mitapivat达到了以血红蛋白应答为指标的主要终点。

DCA will be used with a proprietary genetic test to treat an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD) 1. The FDA granted Priority Review ... DCA is a ...

Pyruknd won its first approval in 2022 for pyruvate kinase (PK) deficiency, marking the first drug approved to treat the disease. The vaccine was previously approved for use in females who are 9 ...