Agios to Present New Mitapivat Data in Rare Blood Disorders at 67th ASH Annual Meeting and Exposition

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that new data on ...
Business Wire

Rocket Pharmaceuticals Presents Positive Clinical Data from Danon Disease, Fanconi Anemia and Pyruvate Kinase Deficiency Programs at the 25th Annual Meeting of the American ...

— RP-A501 for Danon Disease well-tolerated in two pediatric patients with no complement-mediated toxicities reported; efficacy update remains on track for Q3 — — Sustained genetic correction observed ...
Nasdaq

Agios Says CHMP Adopts Positive Opinion For PYRUKYND To Treat Pyruvate Kinase Deficiency In Adults

(RTTNews) - Agios Pharmaceuticals, Inc. (AGIO) announced Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion on ...
Seeking Alpha

Agios Presents Updated PYRUKYND® (mitapivat) Long-term Extension Data Demonstrating Sustained Clinical Benefits in Adults with Pyruvate Kinase (PK) Deficiency at 64th ASH ...

– Data Suggest Long-term Treatment with PYRUKYND ® in Adults with PK Deficiency is Associated with Improvements in Hemoglobin, Iron Overload, Transfusion Burden and Patient-reported Outcomes, ...
BBC on MSN

'I'm only 17 but had over 100 blood transfusions'

A 17-year-old girl who has had more than 100 blood transfusions has told how the donations helped her to "lead a normal life" ...
Business Insider

Agios Submits Marketing Authorisation Application to European Medicines Agency for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency

CAMBRIDGE, Mass., June 28, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for ...
Nasdaq

PYRUKYND® (mitapivat) Approved in the EU for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients

CAMBRIDGE, Mass., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined ...
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Agios Announces Publication of Phase 3 ACTIVATE-T Data in The Lancet Haematology Demonstrating Benefits of PYRUKYND® (mitapivat) for Adults with Pyruvate Kinase Deficiency

CAMBRIDGE, Mass., Aug. 18, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, ...
Business Wire

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare ...