Columns

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

My wife, Jill, and I celebrated our wedding anniversary last month. We like to joke that we don’t keep track of the years because it seems like yesterday when we got married. Some years, as a gift, I like to write her something romantic or funny. This year, I decided…

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

I’ve found that having a parent with Huntington’s disease makes you value every moment with them, even in unfortunate situations. For instance, normally I wouldn’t volunteer to go on a road trip from Massachusetts to Illinois, but I would for my mother. As I’ve previously written, my mom was…

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

“Parting is such sweet sorrow,” Shakespeare once wrote. And don’t I know it. Recently, the big news in my home is we’re moving to the East Coast, as my wife, Jill, and I want to move closer to our daughter, who lives in Boston, and other family…

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

Dear people working to find a cure for Huntington’s disease (HD), I imagine you all have various reasons for seeking a cure. Near the top of your list is probably a love for science and a challenge. Or, perhaps you view HD like George Huntington, whose medical observations of people…

One afternoon in May, I got a call from a number I didn’t recognize, and I rejected it immediately. Given the number of spam calls today, I wouldn’t normally log that in my memory, but then I received a voicemail from the hospital where…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…