News

May 12, 2020May 12, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

News, syndicated

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey ... Read more

May 6, 2020May 6, 2020

Study Calls for Healthcare Restructuring in Italy to Better Suit Needs of Adults With PKU

News

Healthcare services need to be restructured in Italy to better support adults with phenylketonuria (PKU), regardless of whether or not they follow strict diet regimens, a study suggests. The study, ... Read more

April 29, 2020April 29, 2020

PKU Children and Adolescents Show Deficits in Executive Function, Study Finds

News

Children and adolescents with phenylketonuria (PKU) have more extensive deficits in executive function — a set of cognitive skills that help in setting goals and getting things done in daily ... Read more

April 27, 2020April 27, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

News, syndicated

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their ... Read more

April 23, 2020April 23, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

News, syndicated

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called ... Read more

April 22, 2020April 22, 2020

Silica Particles May Be Way of Oral Enzyme Replacement Therapy for PKU

News

A carrier system made of tiny silica particles may be used to deliver a therapeutic enzyme directly to the digestive system, a recent study suggests. Such an oral treatment would ... Read more

April 8, 2020April 8, 2020

Phase 2 Trial of SYNB1618 in PKU Likely to Be Delayed Due to COVID-19

News

A planned Phase 2 clinical trial of Synlogic’s SYNB1618, an investigational treatment for people with phenylketonuria (PKU), is expected to be delayed due to the COVID-19 pandemic, the company said. Although ... Read more

April 6, 2020April 6, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

April 2, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

News, syndicated

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

April 1, 2020April 1, 2020

FDA Grants Orphan Drug Designation to APR-OD031 for PKU Treatment

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to APR-OD031, an investigational treatment for people with phenylketonuria (PKU). This designation is intended to promote the development of ... Read more