FORT LEE, N.J. – Kedrion Biopharma Inc., an international biopharmaceutical company specializing in the research and development, production, and commercialization of plasma-derived therapeutic products used in treating rare and serious diseases, announced today that plasminogen deficiency type 1 (PLGD-1) patients in the U.S. now have access to RYPLAZIM® (plasminogen, human-tvmh)....
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A Keele researcher will embark on a two-year study to identify affordable treatments to help children living with spinal muscular atrophy. Dr Melissa Bowerman, of Keele University’s School of Medicine, has been awarded £99,959 by the Academy of Medical Science’s Springboard grant scheme to continue her research into treatments for...
PEA RIDGE – Kyler Kasper is living on borrowed blood. He has not yet celebrated his second birthday, but Kyler’s parents expect him to reach it, thanks to blood donations from strangers. Kyler was recently diagnosed with Diamond Blackfan anemia, a rare blood disorder that affects about 500 people in...
Brussels, Belgium – Patients are central to the rare disease community on all levels. This sentiment was echoed by representatives from all stakeholder groups at the 2023 World Orphan Drugs Congress which took place in early November 2023 in Barcelona. The Parliament joined the Alexion – AstraZeneca Rare Disease team...
BALTIMORE, Maryland — Researchers at Kennedy Krieger Institute are sharing their expertise on autism spectrum disorder in a medical journal reaching thousands of pediatric professionals worldwide. The journal, Pediatric Clinics, provides the latest clinical information on health and related issues for children and adolescents. The newly released volume is titled...
LEXINGTON, Mass. — Keros Therapeutics, Inc. (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (TGF-ß) family of proteins, today announced that the U.S. Food and...
NEW YORK — Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX) today announced data on the clinical activity of KRX-0401 (perifosine), the Company’s Akt-inhibitor for cancer, as a treatment for advanced renal cell carcinoma. Abstract #5034, entitled, “Phase II study of perifosine in metastatic renal cell carcinoma (RCC) progressing after prior therapy (Rx)...
East Hanover, NJ – This year, New Jersey Health Foundation (NJHF) granted $135,000 in awards to three Kessler Foundation scientists to support pilot research projects that exhibit promising potential. Foundation researchers will conduct studies that expand research in web-based job interview training for youth on the autism spectrum, upper extremity...
East Hanover, NJ – A research scientist at Kessler Foundation has been awarded a highly competitive Mentored Patient-Oriented Research Career Development Award (K23) from the National Institute on Minority Health and Health Disparities, a part of the National Institutes of Health (NIH). This $704,054, five-year grant will support one of...
WINNIPEG — Local medical researchers are optimistic they’ve found the key to unlocking a cure for a rare genetic disorder that affects Hutterite babies. Scientists at the University of Manitoba and the Manitoba Institute of Child Health have discovered that a small change in a gene involved in cell growth,...