Early last week, Agios Pharmaceuticals ("Agios") announced that its treatment Mitapivat received Orphan Drug designation. This orally administered therapy is a pyruvate kinase R (PKR) activator. As Agios has…
Recently, clinical-stage biopharmaceutical company Imara Inc. ("Imara") announced that the first patient was dosed in the Phase 2b Forte clinical trial. The trial is evaluating IMR-687, a PDE9-inhibitor, for…
For years, clinical-stage biopharmaceutical company Fulcrum Therapeutics ("Fulcrum") has worked to develop therapies for patients with rare genetic disorders and diseases. Somewhat recently, their focus was on treating beta thalassemia…
Without treatment, spinal muscular atrophy (SMA) is the top cause of infant or child mortality before age 2. As a result, early detection is crucial in improving patient outcomes. But…
For most drug therapies, researchers test on animal models of a disease before testing on humans. Recently, using microRNA-483, a small RNA molecule, on rat models of PAH reduced…
Recently, researchers from European Molecular Biology Laboratory (EMBL) looked to understand the cause of pulmonary arterial hypertension (PAH). They collaborated with Stanford University through the Life Sciences Alliance. Because…
Saying that COVID-19 has impacted the world might just be an understatement. In fact, COVID-19 changed the way we interact with each other, businesses, and the world around us. But how…
As originally reported in Yahoo Finance, the coronavirus is disrupting many of our expectations of how we planned to live out the next few months. Some frequenters of the hospitals…
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