HRA Pharma Rare Diseases and Celnova Pharma are partnering to meet a major unmet need for rare disease drugs in South America.
treatment News
Bethesda, Md. — HYFTOR® (sirolimus topical gel) 0.2%, indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex (TSC) in adults and pediatric patients 6 years of age and older, is now covered by Medicare in Texas, Florida, New York, and California, among other states. Nobelpharma, a pharmaceutical...
Rare diseases are a serious public health concern in India, with an estimated burden of about 80 to 96 million cases reported annually. Moreover, 70-80% of rare diseases are of genetic nature and thus are asymptomatic. Here are their causes, signs, symptoms and treatment
BOSTON, Mass. — Ratio Therapeutics Inc. (Ratio), a pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment and monitoring, announced today that dosing of the first cohort has been completed in the ATLAS trial, a Phase 1/2 open-label study evaluating the safety, tolerability, and efficacy of the company’s lead...
Dublin, Ireland. Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences. The findings underscore a call to make augmentation therapy accessible to patients with AATD in...
Ann Arbor, Mich. – High-grade glioma, an aggressive form of pediatric and adult brain cancer, is challenging to treat given the tumor location, incidence of recurrence and difficulty for drugs to cross the blood-brain barrier. Researchers from the University of Michigan, Dana Farber Cancer Institute and the Medical University of...
New York, NY – In people with pulmonary arterial hypertension (PAH), adding Winrevair (sotatercept-csrk) to standard therapy was linked to longer survival, fewer safety events, and a lower need for lung transplant compared with standard therapy alone, according to new real-world data. “These findings extend clinical trial evidence, suggesting sotatercept [Winrevair] offers meaningful...
OSAKA, Japan & CAMBRIDGE, Mass. – VPRIV (velaglucerase alfa), an approved enzyme replacement therapy (ERT) for Gaucher disease, appears to be safe and effective in children under 4—a population not included in the clinical trials that supported its approval. A recent U.S. real-world study evaluated VPRIV in a small group...
NORFOLK, Va. — ReAlta Life Sciences, Inc., a mid-stage clinical biotech company dedicated to saving lives by rebalancing the inflammatory response to address life-threatening diseases, today announced enrollment of the first patient in a Phase 2 clinical trial evaluating the safety and efficacy of RLS-0071, an investigational new drug based...
NORFOLK, Va. — ReAlta Life Sciences, Inc. (“ReAlta” or the “Company”), a clinical-stage biopharmaceutical company dedicated to saving lives by rebalancing the inflammatory response to address rare and acute inflammatory diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to RLS-0071 (pegtarazimod) for the treatment of...