Changes to a critical structure within the cell nucleus of spinal nerve cells — the nucleolus — preceded the appearance of other disease markers in people with either familial or sporadic forms of amyotrophic lateral sclerosis (ALS), a study has discovered. …

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

“I’m glad I got the chance to miss you,” I told my husband, Todd, when I got home after a day trip skiing with our children last week. Since Todd has ALS and I am his caregiver, we rarely spend time apart. Now that he is paralyzed, we don’t…

The small difference in treatment responses among amyotrophic lateral sclerosis (ALS) patients receiving NurOwn versus a placebo in a recent clinical trial likely was due to chance, the U.S. Food and Drug Administration (FDA) announced in a press release. Along with slightly more deaths in the NurOwn…

PrimeC is safe and well-tolerated for the treatment of amyotrophic lateral sclerosis (ALS) and showed signs of slowing disease progression in patients, findings from a recently-completed Phase 2a study show. All 15 participants in the NST002 clinical trial (NCT04165850) have chosen to…

Last week, I stepped onto my soapbox (mine has special safety handrails) and voiced my opinion about an issue that’s been bugging me for several years. It’s a problem I share with about 7.5 million other people who have trouble using their voices and cannot get voice-activated devices to…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

“There’s an old joke, um … two elderly women are at a Catskill mountain resort, and one of ’em says, ‘Boy, the food at this place is really terrible.’ The other one says, ‘Yeah, I know — and such small portions.’ Well, that’s essentially how I feel about…

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…

I’m happy to announce I’ve finally resolved a love-hate relationship — with my AFOs. It took persistence, but I can now step into my white, molded plastic ankle-foot orthoses and walk pain-free. That’s important to me because I want to keep walking for as long as possible, and my…