Amydis is developing a test to monitor the buildup of TDP-43 in the eye’s retina of people with amyotrophic lateral sclerosis (ALS), which could offer a noninvasive way to diagnose the neurodegenerative disease sooner and better monitor its progression. The research will be conducted with support from a…

A number of stem cell lines developed to better understand how certain genetic factors might contribute to amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases is now available to scientists worldwide. The cell lines were engineered by researchers at the National Institutes of Health Center for Alzheimer’s and Related…

Long-term use of Radicava (edaravone) is safe and may modestly slow disease progression in people with amyotrophic lateral sclerosis (ALS), according to a small Korean study. Patients also experienced no changes in phrenic nerve function, which is required for the contraction and expansion of the diaphragm muscle. “Further…

Kadimastem has received a U.S. patent covering its investigational cell therapy AstroRx for treating amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The patent, granted by the U.S. Patent and Trademark Office, supports an upcoming Phase 2a clinical trial that’s planned to start in the U.S. next year.

My husband is my primary caregiver. In fact, he’s been my only caregiver for the past 12 years that I’ve been living with ALS. That changed last week, when he needed medical tests that required an overnight stay in the hospital. That’s when we turned to a local home…

Note: This story was updated Aug. 10, 2022, to clarify the U.S. Food and Drug Administration advisory committee voted there is no substantial evidence supporting AMX0035 efficacy. When added to standard of care, both the experimental oral therapy AMX0035 and the U.S.-approved Radicava ORS oral suspension provide comparable…

The Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, the largest collection of de-identified records on amyotrophic lateral sclerosis (ALS) patients in clinical trials, has been updated with records of participants in three Cytokinetics-sponsored studies. Newly added findings derive from a decade of trials involving nearly 600 ALS patients. Those…

There I was, just living each day and minding my own business. I was following all the guidelines from the U.S. Centers for Disease Control and Prevention and wearing a mask everywhere I went, when all of a sudden, BAM! The COVID-19 virus found me. What followed were…

Early use of Clene Nanomedicine’s experimental therapy CNM-Au8 reduces the risk of death by 70% in people with amyotrophic lateral sclerosis (ALS), compared with patients who experienced a nine-month delay in starting treatment. These are the findings of an updated analysis of the RESCUE-ALS Phase 2 trial…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…