Phenylketonuria News

January 13, 2021January 13, 2021

Homology Medicines’ Gene Therapy, Gene Editing Programs Advance

Homology Medicines has announced the next steps for its gene therapy and gene editing clinical programs for the treatment of phenylketonuria (PKU). “We ended 2020 with positive data from the dose-escalation ... Read more

January 6, 2021January 6, 2021

Inaccurate Protein Content Found on Most Food Labels in UK

Phenylketonuria News

Food labels on supermarket websites in the U.K. have high rates of misleading, incomplete, and inaccurate information relating to protein content, which could affect the metabolic control of people with ... Read more

December 16, 2020December 16, 2020

New CRISPR-Cas9-created Pig Model Better Mimics PKU Symptoms in Humans

Phenylketonuria News

Using the CRISPR-Cas9 gene-editing tool, researchers created a pig model of phenylketonuria (PKU) that better mimics the disorder’s signs and symptoms in humans, compared with classic mouse models, a study ... Read more

December 9, 2020December 9, 2020

New Paper-based Sensor Could Help Monitor PKU, Study Finds

Phenylketonuria News

A novel sensor that combines plant enzymes and nanomaterials can be used to measure levels of the amino acid phenylalanine in human blood, a new study shows. The sensor may be ... Read more

December 2, 2020January 3, 2021

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Phenylketonuria News

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) ... Read more

November 26, 2020December 28, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

COVID-19 News, Phenylketonuria News

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a ... Read more

November 25, 2020November 25, 2020

Pfizer Invests in Homology Medicines, Supporting Gene Therapies for PKU

Phenylketonuria News

Homology Medicines, which specializes in gene therapies for rare diseases and is developing two potential such therapies — HMI-102 and HMI-103 — for people with phenylketonuria (PKU), has received a $60 million equity investment from Pfizer. ... Read more

November 20, 2020December 24, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Phenylketonuria News

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how ... Read more

November 18, 2020November 18, 2020

NPKUA Urges Adult Patients to Join PKU Registry, Assist Research

Phenylketonuria News

The National PKU Alliance (NPKUA) is encouraging people with phenylketonuria (PKU) to join the PKU Registry, which is working to help scientists better understand and manage this disease, establish best practices, ... Read more

November 11, 2020November 11, 2020

HMI-102, Potential PKU Gene Therapy, Fares Well in Phase 1/2 pheNIX Trial

Phenylketonuria News

HMI-102, Homology Medicines‘ gene therapy candidate for adults with phenylketonuria (PKU), was well-tolerated and induced marked reductions in phenylalanine and phenylalanine-to-tyrosine ratio (Phe/Tyr ratio) in some patients. Those promising results ... Read more

November 4, 2020December 6, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

Phenylketonuria News

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations ... Read more

October 28, 2020October 28, 2020

Reduced Gray Matter Volume Found in Brain Region of Early-treated PKU Patients

Phenylketonuria News

People with phenylketonuria (PKU) who receive treatment soon after birth still experience significant reductions in the volume of gray matter, which contains mainly nerve cell bodies, in their cerebellum, a ... Read more

October 27, 2020November 28, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Phenylketonuria News

A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in ... Read more

October 21, 2020October 21, 2020

Dr. Reddy’s Launches Generic of Kuvan for PKU Patients in the US

Phenylketonuria News

Dr. Reddy’s Laboratories has launched a generic version of BioMarin Pharmaceuticals’ Kuvan for the treatment of adults and children with phenylketonuria (PKU) in the U.S. This generic will be distributed ... Read more

October 14, 2020October 14, 2020

FDA Approves Use of Higher Palynziq Doses in Adults With PKU

Phenylketonuria News

The U.S. Food and Drug Administration (FDA) has agreed to increase the maximum Palynziq (pegvaliase-pqpz) dose allowed for treating adults with phenylketonuria (PKU), BioMarin announced. While the maximum dose previously ... Read more