rare disease

This year, Thanksgiving felt quite special. For starters, it was the first time I celebrated with both of my kids. My youngest, Kyree, turns 1 three days after Christmas, and so far, his first holiday season has been so much fun. As I reflected over the holiday, I took some…

Time: It seems like we never have enough of it. It goes by so fast when things are great, but when things aren’t so hot, it seems to drag on. We never have enough time to do all the things we want to do, and then one day, it’s gone.

For me, getting diagnosed with late-onset Pompe disease was a relief. Although there’s no cure, treatment is available to help slow disease progression. After starting treatment, I wondered what my life would look like. Would I have to stay home? How long would I be able…

Having a child with a rare disease can certainly be challenging at times, but it doesn’t make us superheroes. We’re often called this by people with typical children or no children at all. The truth is that we’re just like everyone else. We don’t have magical superpowers, nor do…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

In a perfect world, we might expect doctors and nurses to know about every medical issue that could possibly arise. However, this just isn’t possible. Although these professionals go through many years of schooling, they can’t be taught about every disease, especially if it’s a rare one. Over the years,…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

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…