‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…

A Temple University research team received a National Institutes of Health (NIH) grant totaling $1.25 million to explore the molecular underpinnings of neuronal degeneration in amyotrophic lateral sclerosis (ALS) and other diseases. With this five-year grant, the team led by Gareth Thomas, PhD, an associate professor of neural sciences…

I love daily routines. For me, having a set of habits and to-do projects waiting to be tackled helps me feel grounded when the world’s events are in turmoil. And more importantly, daily routines keep my mind focused on things other than my ALS. What I don’t love are disruptions,…

Alexion Pharmaceuticals is stopping further work on Ultomiris (ravulizumab) — its approved therapy for complement-associated rare blood disorders — as a potential treatment of amyotrophic lateral sclerosis (ALS) based on interim data from the CHAMPION-ALS Phase 3 clinical trial. While top-line results were expected by mid-2022, the study’s independent data…

People affected by amyotrophic lateral sclerosis (ALS) have come up with plenty of creative ways to raise money for research and care — whether it’s the ALS Ice Bucket Challenge, re-creating Olympic events, or Major League Baseball’s designation of June 2 as Lou Gehrig Day. Joe…

MediciNova is still recruiting adults with early-stage amyotrophic lateral sclerosis (ALS) for the COMBAT-ALS Phase 2b/3 clinical trial evaluating its investigational oral therapy MN-166 (ibudilast). Patients are being enrolled at 19 sites across North America; more information about contacts and locations is available here. “We continue to…

I pleaded. I bargained. I threatened. But it was no use. My dog would not back away from the woods behind the shed, where he had been sprayed by a skunk just two weeks earlier. I was more panicked when he got skunked than I was now. I frantically searched…

Clene, and its wholly owned subsidiary Clene Nanomedicine, have signed two lease agreements that will more than quadruple the manufacturing capacity of CNM-Au8, their investigational therapy for people with amyotrophic lateral sclerosis (ALS). The first lease is for a 10-year agreement for a 74,210-square-foot building in Elkton, Maryland.

Long-term treatment with Radicava (edaravone) is generally safe and nearly doubles survival time in adults with amyotrophic lateral sclerosis (ALS), according to a small real-life study in Japan. These data add to increasing, but still limited, evidence showing the therapy prolongs the life of ALS patients and…

This past week was filled with voices. No, I wasn’t hearing voices in my head. But everything seemed to involve a voice. Let me explain. An action hero uses his voice I watched the newly released documentary “Val,” currently streaming on Amazon Prime Video. By the time the final…