Otsuka Pharmaceutical is broadening its scope in rare disease, striking a deal to acquire Jnana Therapeutics and its lead drug candidate, a small molecule on track for pivotal testing in a metabolic ...

—Researchers in Germany recently analyzed several parameters related to bone metabolism in plasma, serum, and urine—as well as tracked dietary habits and lifestyle factors—in a cohort of adults with ...

—According to a new systematic review of the literature, despite early and continuous dietary management to lower phenylalanine levels in patients with phenylketonuria (PKU), cognition may still be ...

The U.S. Food and Drug Administration has approved Sephience (sepiapterin), an oral therapy by PTC Therapeutics, for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder that ...

Phase 1 data in healthy volunteers exceed expectations and support best-in-class potential, enabling Phase 2 advancement for both intended indications of PKU and CKD Dose-dependent urinary amino acid ...

(RTTNews) - PTC Therapeutics, Inc. (PTCT) Monday said that the European Commission has granted marketing authorization for Sephience for the treatment of phenylketonuria (PKU). The company noted that ...