We are joined today by Dr. Stephen Goutman, an associate professor of neurology at the University of Michigan and the director of Michigan Medicine’s ALS Center of Excellence and Multidisciplinary ALS Clinic. The clinic was recently awarded an ALS Association Clinical Management grant to support Dr. Goutman’s important study aimed at improving non-invasive ventilation for people with ALS through the use of custom-printed 3D masks.
Today, we are happy to be joined by Dr. Megan McCain, an assistant professor of Biomedical Engineering and Stem Cell Biology and Regenerative Medicine at the University of Southern California (USC), an ALS researcher who recently received an investigator-initiated starter grant award from The ALS Association. These awards are designed to help bright researchers start their own labs to answer their own innovative questions addressing ALS disease.
Assistive technology can provide a major benefit to people living with ALS and their caregivers. That is why some of the advances we have seen in recent years, thanks to generous donations from the ALS Ice Bucket Challenge and beyond, are so exciting.
Researchers funded by The ALS Association, through donations from the ALS Ice Bucket Challenge, discovered new evidence on the role that mutant TDP-43 plays in development and progression of the disease. This important work sheds light on novel aspects of TDP-43 biology and provides valuable tools to gain insight into early stages of ALS disease progression and could lead to the development of new therapies.
The ALS Association is hosting ALS Community Workshop: Developing Drugs for Treatment, Guidance for Industry on July 12 in Washington, D.C., and the entire ALS community is invited. The goal of the workshop will be to provide targeted feedback and information to the Food and Drug Administration (FDA) from people with ALS, caregivers, and stakeholders to inform revisions of the FDA’s Draft Guidance on ALS Drug Development.
Erin Brady Worsham, an award-winning artist based in Nashville who has lived with ALS for more than 24 years, takes her philosophy about life from golf.
In recognition of ALS Awareness Month, we’ve been sharing a lot of new stories about people living with the disease and their caregivers, the volunteers and health care providers who help make the lives of people with ALS better, the powerful fundraisers and the participants in Walk to Defeat ALS and Team Challenge ALS events, and the researchers fighting for a cure. This is the 14th profile in that series.
“I raise my voice for the people who can’t. Someone I love was affected by ALS, so it affected me as well. I say what I [feel] the people who lost their communication abilities would want to be said,” 13-year-old Sophie Rubin said.
Treeway, a biotech company in the Netherlands with a mission to develop a cure for ALS, recently announced promising results of its phase I trial of its lead drug, TW001. TW001 is an oral formulation of edaravone. Radicava®, the most recently FDA approved drug to treat ALS, is an intravenous version of edaravone. The study showed that TW001 was well tolerated and stable, while improving bioavailability compared to Radicava.
Bad things happen to good people every day. That’s just the nature of life. But it doesn’t mean we stop living and loving. My ALS story officially started on a warm fall afternoon in September 2001. I had been having difficulties with speaking and swallowing for several months. After more than two months of seeing about a dozen different medical professionals, and having more tests than one can imagine, I sat in the small examination room of a local neurologist.
Margot Vaughan has been fighting ALS for more than two years. But she hasn’t been fighting alone. “My family is totally devoted to me,” Margot said. “They keep me going and I’m lucky and grateful to have such an amazing family by my side.”