My husband, Jared, and I have an open-door policy for our friends. We warmly welcome them, whether they need a place to stay for the night, a safe haven after a tough experience, a spot to relax, or a place to celebrate life’s victories. In our home, friends become part…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

Laughable. That’s how Heidi Bentz and Lucrecia E. Choto, two women who struggled for years to receive a proper diagnosis of von Willebrand disease (VWD), described the results of a 2004 survey by the U.S. Centers for Disease Control and Prevention (CDC) of 75 women who…

If you are active on social media and have a chronic illness, you’ve probably come across the term “spoonie.” The word comes from a woman’s explanation of how she has to manage her energy levels each day.  As she was in a cafe at the time, she grabbed a bunch of spoons…

My husband, Jared, and I have been fur parents for a while. In 2020, we welcomed our first family pet, a golden retriever named Lucky, followed by a delightful Himalayan cat we named Billy. Our home has been lively with the presence of animals for the past three years.

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Modern psychology often emphasizes the importance of letting go. In a world constantly pushing us to hold on tightly, learning to let go can be surprisingly liberating. As I get older, I’m realizing the significance of discerning what truly matters in life and cutting out the fluff. Our capacity…

After handing over the development of the gene therapy candidate SB-525 to Pfizer, Sangamo Therapeutics has now concluded its collaboration agreement with Biogen to advance and market gene therapies for Alzheimer’s and Parkinson’s diseases, and other neuromuscular and other neurological disorders. Among other goals, the…

What’s new, month of May? Why hello there, extended coronavirus lockdown!   Our community quarantine was set to end on April 30. In the last few days of April, I felt so excited to go back to how life was before COVID-19. I missed going out, eating…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…