Mary Chapman, features writer —

Replagal is a treatment for Fabry disease patients ages 7 and older. Shire (now owned by Takeda) developed it. Although in use in many countries, including those of the European Union, the U.S. Food and Drug Administration (FDA) has not approved Replagal. Following are answers to some frequently…

Emotional resilience is the ability to adapt to stressful situations and cope with life’s ups and downs. Here’s how you can develop emotional resilience and use it to help you in your journey with Fabry disease. What is Fabry disease? Fabry disease is a progressive rare genetic disease…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

A Japanese concept called shoshin might help people with diseases such as Fabry disease change their perspective and see their world, and their disorder, in a different light. What is Fabry disease? Fabry disease is a progressive and rare genetic disease. It results from the abnormal buildup of a…

If you or a family member has a rare disorder such as Fabry disease, you may want to get involved with health advocacy efforts. In general, these efforts promote health and access to care at individual and community levels. Here is more information about advocacy and ways in which…

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

The annual EURORDIS Photo Award contest is inviting people worldwide to visually express what life is like with a rare disease, while raising awareness through their work of these disorders. Submissions for next year’s awards are open until Jan. 31. The competition is organized by France-based EURODIS, an alliance…