With apologies to Jimmy Page and Robert Plant: Chairway to Heaven I can no longer walk down the road Unable to take on any load Those facts cause much grievous woe…

A new molecular mechanism that contributes to the death of some nerve cells has been discovered by a team of scientists led by researchers at Oregon State University. The researchers found that blocking a protein called HSP90 can result in the activation of a receptor present in motor…

After we got married, Todd and I bought an adorable, craftsman-style bungalow in a Milwaukee neighborhood. I believed living in the city would give me a better understanding of the issues faced by the families I served at an inner-city ministry, where I coordinated a tutoring program. Our next-door…

Mutations in the FUS gene, one of the most common causes of familial amyotrophic lateral sclerosis (ALS), increase the number of branches extending from the axons of motor nerve cells, so that no clear path exists for these neuronal “arms” to transmit information — in the form of electrical impulses —…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

“So tell us your story,” we often ask people who come to help with Todd’s care. Those who show particularly deep compassion tend to have their own story of suffering, or they’ve loved and cared for someone who suffered. A stage IV cancer survivor. A disabled parent. A sibling who…

Orphazyme announced that it and Worldwide Clinical Trials will continue their collaboration through the long-term extension of a Phase 3 study into arimoclomol, a potential oral treatment for amyotrophic lateral sclerosis (ALS). ORARIALS-01 is a randomized, placebo-controlled and double-blind trial (NCT03491462) underway at 30 centers across North America…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

A few years ago, during an ALS clinic visit, the topic of voice banking came up. I was assured that it was a way to help me to prolong my ability to communicate. Banking my voice? I’d never heard of it. So, I left with instructions to check…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…