A Chiesi Farmaceutici drug for a rare enzyme deficiency is now approved by the FDA, making it the first U.S. treatment for a disorder that leads to a range of cognitive and muscle problems. The ...
A drug developed by partners Protalix BioTherapeutics and Chiesi Group has won FDA approval, a regulatory decision that follows the product’s European approval last week, introducing new competition ...
Saol Therapeutics received a complete response letter for its pyruvate dehydrogenase complex deficiency treatment a week ...
In certain groups of men, glucose-6-phosphate dehydrogenase (G6PD) deficiency was linked with an increased likelihood of severe COVID-19, a Veterans Affairs cohort study found. In Black men under 65, ...
A parliamentary forum has urged urgent action on funding shortfalls and treatment delays for patients with Lysosomal Storage ...
Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ...
Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder. It can occur in anyone but is more common in people with ancestry in North and Central Europe. People with AATD experience ...
Ischemic heart disease, the leading cause of death in the United States, has long puzzled researchers. While high serum cholesterol levels are linked to cardiovascular risk, the role of dietary fat ...
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