Saol Therapeutics hit with CRL for rare genetic disease
The U.S. FDA issued a complete response letter (CRL) for the NDA to privately held Saol Therapeutics Inc.’s…
Medicines Reimagined – Repurposing Drugs For New Indications
In a group of health-related headlines, one says “Hope for new drug approach. getty As government-supported research in…
Ionis gets approval for preventing attacks in rare and genetic HAE
In Ionis Pharmaceuticals Inc.’s second U.S. FDA approval in under a year, the agency approved Dawnzera (donidalorsen) as…
Bridging psychiatry and rare genetic diseases: a scoping review of therapeutic strategies and diagnostic delay paired with healthcare economic burden analysis | Orphanet Journal of Rare Diseases
Nguengang Wakap S, Lambert DM, Olry A, Rodwell C, Gueydan C, Lanneau V, Murphy D, Le Cam Y,…
Researchers classify how specific genetic mutations correspond to rare disorders
The NF-κB signaling pathway plays a pivotal role in how our bodies fight infection, heal, and regulate inflammation.…
Heart Failure, AF, and Mortality More Common After RSV Than UTI or Flu Hospitalizations in Older Adults
Chris Verschoor, PhD, HSN Credit: Health Sciences North Research Institute Respiratory syncytial virus (RSV) hospitalizations led to poorer…
UConn, Connecticut Children’s trial gene-editing for rare disease
Julieta Bonvin Sallago is constantly plagued by the worry that she will not wake up in the middle…
AI Tool Interprets Echocardiograms in Minutes, New Yale Study Finds
Rohan Khera, MD, MS Credit: Yale University Cardiologists use echocardiography to diagnose a range of functional or structural…