WebFeb 5, 2024 · Treatment of IEMs has traditionally relied on treating the nutritional and/or metabolic environment, for example, replacement enzyme therapy, rather than the genetic disorder itself. Rapid advances in gene therapy and its safety may one day be possible. As of 2024, phase 1, gene-therapy trials for PKU remain in the planning stage. WebJun 2, 2024 · “The impacts of phenylketonuria stretch further than simply dietary concerns, and this therapy offers great promise to these individuals to possibly having a better quality of life, throughout ...
What are common treatments for phenylketonuria (PKU)?
WebMay 16, 2024 · Part 1 of this study will evaluate the safety and efficacy of HMI-102 gene therapy in adult subjects with PKU due to PAH deficiency. Subjects will receive a single … WebMay 24, 2024. The U.S. Food and Drug Administration today approved Palynziq (pegvaliase-pqpz) for adults with a rare and serious genetic disease known as … human gives birth
Long-Term Metabolic Correction of Phenylketonuria by AAV ... - PubMed
WebNational Center for Biotechnology Information WebSep 6, 2024 · Homology Medicines is developing HMI-102, as an investigational gene therapy to treat phenylketonuria (PKU) in adults. The therapy is designed to encode the PAH gene, which is mutated in people with PKU, delivered via the liver-tropic AAVHSC15 vector. In May 2024, the US FDA granted Fast Track designation for HMI-102, … WebJul 15, 2024 · The gene therapy uses an adeno-associated viral vector (virus engineered to be harmless) to shuttle a fully functional copy of the PAH enzyme gene into the liver cells … human givens training courses