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…
Passive Smoke Exposure in Prepubescent Males Affects Lung Function in Children, Grandchildren
A new study has found that paternal prepubertal passive smoke exposure was associated with impaired lung function trajectories…
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…
Rising Obesity Rates in Adolescents, Young Adults Tied to Major Health Complications
Ashwin Chetty Credit: Yale School of Medicine New research is shedding light on large portions of obesity-related complications…
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,…
DGBI Burden, Prevalence Increased After COVID-19 Pandemic, Study Finds
Olafur Palsson, PsyD Credit: Olafur Palsson on LinkedIn New research is shedding light on notable increases in the…
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.…
GLP-1 RAs More Effective Than Metformin for Reducing Dementia Risk in T2DM
Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) may be more effective than metformin for reducing the risk of dementia…
Child walks again after experimental treatment for genetic condition
In what experts are calling a “dream come true,” scientists used a recent biochemical discovery to help an…
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…