FDA Grants Orphan Drug Status to Amylyx’s AMX0035 for Treatment of ALS

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals, the drug developer, announced in a press release. Orphan drug status aims to encourage therapies for rare and serious diseases through incentives such as seven years of market…

Managing a chronic illness can be difficult. There are many different medications to take (often at different times), appointments to remember, symptoms to keep track of, and lots of information to absorb. Thankfully, living in a digital age means that there are numerous mobile apps that can help you manage your chronic…

A mechanism that cells normally use to keep them healthy was found to have a dual role in amyotrophic lateral sclerosis (ALS). While the process, called autophagy, delays disease progression in early stages, it contributes to ALS’s deadly spread through the spinal cord in later stages. This study, “…

Dear readers, this week my Living Well with ALS column will take a slightly different path down the road of wellness. Because, in the quest of living a balanced life, I’ve learned that humor is something we all need to incorporate into our days. And one of…

Neuroscientist and professor Niels Birbaumer, a senior research fellow at Switzerland’s Wyss Center in Geneva, has written a new book investigating the brain’s seemingly limitless capacity  to reshape itself and overcome disease. Birbaumer has conducted research with amyotrophic lateral sclerosis (ALS) patients who, because of their progressive motor neuron disease, have…

Severe DNA damage and other disease-associated hallmarks are absent in a mouse model of familial amyothrophic lateral sclerosis (fALS), a new study shows. The study, “DNA strand breaks and TDP-43 mislocation are absent in the murine hSOD1G93A model of amyotrophic lateral sclerosis in vivo and in vitro,” appeared in the journal…

Welcome to “The Mighty Mind.” When I was diagnosed with ALS at age 28 in 2015, I felt like I had lost everything. My body, without warning or reason, had turned on me, and that meant the end of so much. My teaching career, and dreams of motherhood and growing…

The investigational gene therapy VM202, developed by VM BioPharma and intended for the treatment of amyotrophic lateral sclerosis (ALS), has been shown to be safe and well-tolerated in a small Phase 1/2 clinical trial, according to data published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. While the study,…

The National Institutes of Health has awarded $30 million over two years to 13 researchers who are developing three-dimensional models of amyotrophic lateral sclerosis and other diseases. Researchers say the initiative is aimed at tackling a translational science problem: More than 60 percent of therapies prove ineffective in clinical trials of…

In this video from CBS Sunday Morning, follow the journey of award-winning filmmaker Simon Fitzmaurice as he completes his pet project, a film called My Name is Emily, despite being diagnosed with amyotrophic lateral sclerosis (ALS) eight years ago. MORE: Explaining the progression…