The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Apic Bio‘s APB-102, an investigational gene therapy aiming to treat familial amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. “This orphan drug designation represents an important…
Cognitive problems have limited impact on overall health-related quality of life (QoL) in people with amyotrophic lateral sclerosis (ALS) — but verbal fluency and space orientation are among the factors linked to patients’ emotional well-being, according to new research. The study, “Cognitive deficits have only…
While rehabilitation programs that include exercise are helpful to amyotrophic lateral sclerosis (ALS) patients, no extra benefit was seen by raising the number of weekly sessions from a standard two to five, a small study from Italy reports. The study, “High‐frequency motor rehabilitation in amyotrophic lateral sclerosis: a randomized…
When I was brainstorming ideas for the name of this column, before settling on “Joyful Sorrow,” I asked my husband if he had any ideas. “How about ‘The Caregiver’?” Todd suggested. I dismissed it out of hand. He pushed back. “I think it’s a good name. It’s descriptive and it’s…
The gut microbiome — the natural collection of microorganisms living in our guts — might influence the progression of amyotrophic lateral sclerosis (ALS), recent research suggests. Specifically, researchers found that a metabolite produced by the Akkermansia muciniphila bacteria, called nicotinamide, slowed disease progression and prolonged survival in an ALS mouse…
I always have had a fascination with words. It stems from my mom and dad, both of whom instilled in me a voracious appetite for reading and writing. Letters from my grandfather blending humor, pathos, self-deprecation, irreverence, sobriety, fact, and thoughtful opinion cemented the notion that words, carefully pieced together,…
Concerns over an active pharmaceutical ingredient used in a 2017 study of Nurtec (riluzole) as a bioequivalent medicine were voiced by the U.S. Food and Drug Administration (FDA) in its response to a request for the treatment’s approval for amyotrophic lateral sclerosis (ALS), its developer, Biohaven Pharmaceuticals,…
In 2010, a few short months after my ALS diagnosis, I found myself having to rely full time on a rollator. I’ll be the first to admit I wasn’t happy at all. But now, nine years later, it’s become my ever-present silent buddy, and I can’t imagine my life…
The Chinese caterpillar fungus (Hirsutella sinensis), used in traditional Chinese medicine, minimized motor neuron loss and extended the lifespan in a mouse model of amyotrophic lateral sclerosis (ALS), a study has found. The study, “Therapeutic effects of hirsutella sinensis on the disease onset and progression of amyotrophic lateral…
Patient enrollment is now complete, ahead of schedule, for the Phase 3 ORARIALS-01 clinical trial assessing the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). “The completion of enrollment for Orphazyme’s pivotal ALS trial is another great milestone for our clinical development program. We are confident that…
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