Salt Lake City, Utah – Some families call it a trial of faith. Others just call it a curse. The progressive neurological disease known as spinocerebellar ataxia 4 (SCA4) is a rare condition, but its effects on patients and their families can be severe. For most people, the first sign...
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Baltimore, Maryland – Age-related changes that cause the skin to stiffen and become less elastic may also contribute to higher rates of metastatic skin cancer in older people, according to research by investigators from the Johns Hopkins Kimmel Cancer Center. The study, published March 12 in Nature Aging, shows that increased...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported a full analysis of updated data, including patient-reported outcome (PRO) measures, from its global Phase 3 ACTIVATE and ACTIVATE-T studies of mitapivat in adults with pyruvate kinase...
Cambridge, Mass. – Rare disease company Agios Pharmaceuticals reached the primary endpoint in a Phase III study for its drug pyrukynd (mitapivat) in adults with non-transfusion-dependent alpha- or beta-thalassemia, according to the company’s announcement on Wednesday The Phase III ENERGIZE study met the primary endpoint of hemoglobin response, defined as...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced positive results from its Phase 2, open-label, multicenter study of mitapivat in adults with non-transfusion dependent . Data from the study will be featured in an oral...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, today announced it will sponsor Citizen Scientist: Unpacking the Science of Sickle Cell Disease, an initiative developed by the Sickle Cell Community Consortium (SCCC) to promote health...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, announced that it has submitted a New Drug Application (NDA) for mitapivat to the U.S. Food and Drug Administration (FDA) for the treatment of adults...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, today announced that it has submitted a Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for TIBSOVO® (ivosidenib tablets) as a potential...
Cambridge — Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, announced that the company is scheduled to present at the following March investor conferences: Cowen 43rd Annual Health Care Conference on Monday, March 6, 2023 at 1:30 p.m. ET; and...
GAINESVILLE, Fla. and CAMBRIDGE, Mass. – Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has initiated plans to lease a build-to-suit 21,000 square foot current Good Manufacturing Practices (cGMP)...