SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data from the first study on the impact of caring for a child with the ultra-rare genetic disorder, aromatic L-Amino acid decarboxylase deficiency (AADC-d). Results show that caregivers spend almost every waking moment caring for a child with AADC-d,...
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SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive early results from an investigator-sponsored Phase 2 study of VOXZOGO® (vosoritide) in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Toronto, Canada, March 12-16, 2024....
STOCKHOLM–– Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) today announced that its commercial partner Everest Medicines (HKEX 1952.HK) received approval from the Pharmaceutical Administration Bureau of the Macau Special Administrative Region, China. The approval for Nefecon is for the treatment of primary immunoglobulin A nephropathy (IgAN) in adults at risk...
A drug combination can safely prevent transplanted stem cells (graft) from attacking the recipient’s (host) body, allowing them to develop into healthy new blood and immune cells, a new study shows. Researchers say stem cell transplantation, especially from members of the same family, has transformed the treatment of leukemia, a...
Bristol, England – New research has shown a new type of inhibitor drug could prevent microvascular diabetic complications, such as diabetic eye and kidney disease. The University of Bristol-led research is published in Cardiovascular Diabetology. Diabetes, a disease which results in uncontrolled blood glucose levels, is estimated to affect one...
Dietary management drugs have transformed Type 2 diabetes care, but daily injection routines are challenging for some patients. A new hydrogel could mean shots just three times a year. Stanford, CA – Materials engineers at Stanford University have developed a novel hydrogel drug delivery system that transforms daily or...
Turku, Finland – Cutaneous squamous cell carcinoma (cSCC), also known as the squamous cell carcinoma of the skin, is the most common form of metastatic skin cancer. The incidence of cSCC is increasing worldwide with millions of new cases diagnosed each year. The prognosis of metastatic cSCC is poor as...
TMC207 is a safe and effective drug for the treatment of multidrug-resistant tuberculosis (TB), the results of a randomised, placebo-controlled trial published in the June 4th edition of the New England Journal of Medicine have shown. Patients who received the drug were significantly more likely to have a negative culture...
SAN DIEGO – Non-Hispanic Black patients with acute myeloid leukemia (AML) are living longer, now that new therapies are available, according to a study presented by researchers from the University of Pennsylvania Perelman School of Medicine and Penn Medicine’s Abramson Cancer Center at the 65th American Society of Hematology (ASH)...
Scientists have discovered in more detail than ever before how the human body’s immune system reacts to malaria and sickle cell disease. The researchers from the universities of Aberdeen, Edinburgh, Exeter and Imperial College, London have published their findings in Nature Communications. Every year there are ~200 million cases of...