The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

The pivotal Phase 3 clinical trial testing BrainStorm Cell Therapeutic’s cell-based therapy NurOwn in amyotrophic lateral sclerosis (ALS) patients continues with only occasional scheduling changes to treatment due to the COVID-19 pandemic. Patient dosing is expected to be complete by July, the company announced. “BrainStorm made…

I cried buckets in the months after my husband, Todd, was diagnosed with ALS. When I met with a counselor, I described my overwhelming sadness. “It is sad,” he agreed. “When you read stories, they have happy parts and sad parts. This is a sad part of your story.”…

The hubbub percolating around the SpaceX launch last weekend reminded me of the role a committed and engaged support team plays in averting and staving off peril.  In any successful outcome to a human hostage confrontation, two common strategies unfold. First,…

On Memorial Day, my mom, the kids, and I visited Lakeside Cemetery in Hancock, Michigan. My mom and the kids left in time to attend the Memorial Day service. I arrived late, just as it concluded, because I needed to help Todd in the bathroom and then…

Mitsubishi Tanabe Pharma America (MTPA) has set up a support program for adults living with amyotrophic lateral sclerosis (ALS) and wanting to learn more about this disease and Radicava (edaravone), an intravenous ALS treatment the company markets in the U.S. Called JourneyMate, the program connects ALS patients…

A large part of successfully living with ALS is to face each challenge with a positive mindset and find workable solutions so I can continue to move forward with my life. Lately, a 4-by-6-inch piece of cloth — yes, a face mask — is putting me to the test. What’s…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

A few years before my husband, Todd, was diagnosed with ALS, he baked a cake for a church fundraiser with one hand while our infant daughter slept on her stomach across his other forearm, her head cradled in his hand. It was impressive enough that he could make the traditional…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…