ALS Combination Therapy PrimeC Receives FDA’s Orphan Drug Designation, Enters Clinical Trials

PrimeC, a new combination treatment by NeuroSense Therapeutics to slow down or halt the progression of amyotrophic lateral sclerosis (ALS), has received orphan drug status from the U.S. Food and Drug Administration (FDA), and is being investigated in two recently-initiated clinical trials. The decision to…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

Sometimes you get lucky and life gives you a break, which is not always the norm for someone living with ALS. The past two weeks have been rather fun for me, thanks to a suggestion that I begin using a portable microphone. I have written about my challenges…

A new spinal cell delivery method may reduce the risks and boost the effectiveness of stem cell therapy designed to regenerate the nervous system in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), an early study in rats has found. The study, “Spinal parenchymal occupation by…

Much work is ongoing in amyotrophic lateral sclerosis (ALS), as researchers look for better ways of treating people with a disease that progressively cripples and kills motor neurons — and possibly finding a first therapy addressing the root cause of ALS rather than its symptoms. “ALS is always…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

A month after my husband, Todd, was diagnosed with ALS, I wrote in my journal: “Squeezing in memories is bittersweet. Last weekend, we took Sara and Isaac to the beach. The sun sparkled off the water and Isaac pointed at the seagulls flying over Lake Michigan. I took pictures…

A new tool to search for medicines that might treat neurodegenerative diseases linked to problems with mitochondria, including amyotrophic lateral sclerosis (ALS), has been developed by a team of scientists at the Scripps Research Institute, a study reports.  This tool…

At the time of his stunning 2015 amyotrophic lateral sclerosis (ALS) diagnosis, Ed Rapp was a veteran Caterpillar executive who had worked and lived around the world. Since being diagnosed, Rapp has directed his efforts toward ALS advocacy. Those efforts will be recognized on Feb. 22, when he…

A single protein called Gemin3 may be the bridge linking the varied proteins known to underlie amyotrophic lateral sclerosis (ALS), suggesting a common pathway in this disease, a study using a fruit fly model showed.  The discovery of this common…