Updates

October 28, 2020October 28, 2020
UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front ... Read more
October 27, 2020October 27, 2020
Chiesi Global Launches ‘Rethink Fabry’ Campaign With New Resources, Website

Chiesi Global Rare Diseases has launched a campaign called “Rethink Fabry” that seeks to help people with the rare genetic disorder, along with their ... Read more
October 23, 2020October 23, 2020
Fabry Disease and Anesthesia

People with Fabry disease may need, at some point, to undergo medical procedures that require anesthesia. Here is information about the different types of ... Read more
October 22, 2020October 21, 2020
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its ... Read more
October 20, 2020October 20, 2020
Protalix, Chiesi Launch Expanded Access Program for PRX-102 in the US

Protalix BioTherapeutics and Chiesi Global Rare Diseases have launched an expanded access program (EAP) in the U.S. for PRX-102 (pegunigalsidase alfa), an investigational enzyme ... Read more

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February 6, 2018February 6, 2018

Protalix’s Pegunigalsidase Alfa Granted FDA Fast Track Status for Fabry Disease

News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to pegunigalsidase alfa, or PRX-102, Protalix BioTherapeutics’ therapy candidate under development for Fabry disease. FDA’s fast track status is designed to facilitate the development and speed up FDA review ... Read more

Updates

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

Chiesi Global Launches ‘Rethink Fabry’ Campaign With New Resources, Website

Fabry Disease and Anesthesia

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Protalix, Chiesi Launch Expanded Access Program for PRX-102 in the US

Fabrazyme vs. Replagal for the Treatment of Fabry Disease

Kidney Foundation Opens Effort to Raise Awareness of Fabry Disease

Independent Living for Fabry Disease Patients

The Challenges of Parenting With Fabry Disease

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

Preimplantation Genetic Diagnosis (PGD) for Fabry Disease

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

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

Reproductive Options for People With Fabry Disease

My Childhood Made Sense After My Diagnosis

Prenatal Genetic Diagnosis for Fabry Disease

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

Newborn Screening for Fabry Disease

Activities Underway for Newborn Screening Awareness Month

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

FDA Reviewing Application of PRX-102 as Fabry Treatment

Same But Different Photo Contest Celebrates People With Rare Diseases

Raising Awareness of Fabry Disease

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

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

What Do Blood Test Results Mean in Fabry Disease?

Managing Fabry Disease Requires a Balancing Act

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Mindfulness for Fabry Disease Patients

Pain, Negative Health Perceptions Linked to Patient Depression, Study Finds

Protalix’s Pegunigalsidase Alfa Granted FDA Fast Track Status for Fabry Disease