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.…