During a routine test, Johnny's middle school nurse discovered his severe scoliosis, a prevalent symptom of Marfan. This unexpectedly set his family on a path to uncover his underlying diagnosis at ...

Race may play a role in the waitlist experience, allograft quality and transplant outcomes of patients with autosomal ...

A team led by University of Pittsburgh School of Public Health geneticists has shown, for the first time, that a gene "silencer" that resides in junk DNA is directly sparing people from a devastating ...

including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.

They also see three other Phase 3 trials in advanced stages, and these trials target conditions like limb-girdle muscular dystrophy type 2I/R9, autosomal dominant hypocalcemia type 1, and ...

APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.

autosomal dominant, late-onset degenerative muscle disorder presenting in patients at 40-60 years of age. OPMD is principally characterized by severe progressive dysphagia, impacting 97% of ...

The broker notes that a US competitor recently announced positive Phase 1b study data for another gene therapy targeting autosomal dominant polycystic kidney disease (ADPKD). The broker says ...

"Excellent finish to the year with strong growth and high profitability" Fourth quarter and full year summary October - December Total revenues grew 48% (44% at CER1) to SEK 553 (375) million Sales ...