News

October 6, 2020October 6, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

News, syndicated

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare ... Read more

September 29, 2020September 29, 2020

Using Telemedicine Ensures Patient Monitoring and Safety in Emergencies, Study Says

News

In times of emergency, telemedicine, home-based treatments, and lab tests should be adopted whenever possible to help people with Fabry disease manage their condition while ensuring their safety, an ... Read more

September 28, 2020September 28, 2020

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

News, syndicated

It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they ... Read more

September 15, 2020September 15, 2020

Long-Term Galafold Safe, Effective as Fabry Treatment, ATTRACT Extension Shows

News

The results from a one-year extension study of the Phase 3 ATTRACT trial demonstrate the long-term safety and effectiveness of Galafold (migalastat) in people with Fabry disease with specific mutations. ... Read more

September 9, 2020September 9, 2020

Activities Underway for Newborn Screening Awareness Month

News

As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. ... Read more

September 8, 2020September 8, 2020

ST-920 Gene Therapy Found Safe, Effective in Mouse Model; Supports First-In-Human Trial

News

A single administration of the experimental gene therapy ST-920 in a Fabry disease mouse model safely and effectively increased the levels of the alpha-GalA enzyme — which is lacking ... Read more

September 1, 2020September 9, 2020

FDA Reviewing Application of PRX-102 as Fabry Treatment

News

Note: This story was updated Sept. 9, 2020, to reflect that PRX-102 is licensed to Chiesi for all markets, including the U.S. The U.S. Food and Drug Administration (FDA) has ... Read more

September 1, 2020September 1, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

August 25, 2020August 25, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

News, syndicated

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

August 25, 2020August 25, 2020

FDA Puts Fabry Gene Therapy 4D-310 on Fast Track

News

4D-310, a new gene therapy for Fabry disease that is being developed by 4D Molecular Therapeutics (4DMT), has been granted fast track designation by the U.S. Food and Drug Administration ... Read more