Related
FDA Issues Says It Won’t Approve Incyte’s Ruxolitinib Extended Release Tablets to Treat Rare Blood Disorders
Rare Daily Staff The U.S. Food and Drug Administration has issued to Incyte a complete response letter […]
Read moreAddressing the Barriers to Patient Participation in Clinical Trials
A significant obstacle to getting patients to participate in rare disease clinical trials, particularly children, is the […]
Read moreGovernment of Canada Improves Access to Affordable and Effective Drugs for Rare Diseases
Rare Daily Staff The Canadian government said it would invest $1.1 billion (CAN$1.5 billion) over three years […]
Read moreModerna and Generation Bio Enter Strategic Collaboration to Develop Non-Viral Genetic Medicines
Rare Daily Staff Messenger RNA pioneer Moderna has entered into a strategic collaboration with genetic medicines developer […]
Read moreFDA Approves Incyte’s Zynyz for the Treatment of Rare Cancer
Rare Daily Staff The U.S. Food and Drug Administration has approved Incyte’s Zynyz, a humanized monoclonal antibody […]
Read moreFDA Grants Orphan Drug and Rare Pediatric Designations for Dyne’s Treatment for Duchenne Muscular Dystrophy
Rare Daily Staff The U.S. Food and Drug Administration has granted orphan drug and rare pediatric disease […]
Read moreAcer Therapeutics Raises $2.7 Million in Registered Direct Offering Priced At-the-Market
Rare Daily Staff Rare disease drug developer Acer Therapeutics said it is raising $2.675 million in a […]
Read moreQuince Gets Unsolicited Acquisition Offer from Echo Lake Capital
Rare Daily Staff Rare disease therapeutics developer Quince Therapeutics confirmed that it received an unsolicited offer to […]
Read moreNovartis Reports Long-Term Efficacy of Zolgensma
Rare Daily Staff Novartis presented new data from two-long terms studies of Zolgensma, its one-time gene therapy […]
Read more