Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine cancer (PNET) — the same disease that killed Apple’s founder and CEO Steve Jobs. After three years of progress…

Researchers in Germany studying standards of care in amyotrophic lateral sclerosis (ALS) patients found a high unmet need for assistive technology devices. The cohort study, “Provision of assistive technology devices among people with ALS in Germany: a platform-case management approach,” was published in the journal Amyotrophic Lateral Sclerosis and…

Last month, I read a blog post from the ALS Association that made me sit up and pay attention. It told the story of a woman with ALS-related voice issues who learned specific vocal techniques normally utilized by actors to extend her ability to speak without assistive…

Amyotrophic lateral sclerosis (ALS) is sometimes known as Lou Gehrig disease after the baseball player who famously suffered from it. Although ALS is a more common disease than we would like it to be, there is still little known about it and many are unaware of the details of the condition. MORE: Find out…

Restoring a protein that’s missing in a type of ALS that affects 10 percent of patients stopped nerve cell deterioration, University of Southern California researchers report. The results also applied to a disease called frontotemporal dementia, or FTD, according to the team at USC Stem Cell. Their study, ”Haploinsufficiency leads…

Being diagnosed with amyotrophic lateral sclerosis (ALS) is a difficult and stressful time. You’ll probably feel incredibly overwhelmed in the first few days and weeks, and struggle to understand the ramifications involved. To help with the process, we’ve compiled a list of tips for patients who have recently been diagnosed with…

Researchers at Sweden’s University of Gothenburg say low red blood cell count at a young age is a risk factor for amyotrophic lateral sclerosis (ALS). Disease onset was also associated with low muscle strength and low body fat during the later teen years. ”One should never overstate conclusions from a…

https://vimeo.com/124559355 This video from the ALS Association Texas chapter is all about a talented young artist, Alexis. Alexis has loved drawing since she was a child, but the progression of her amyotrophic lateral sclerosis (ALS) condition has meant that she can now no longer draw with paper and pencils. However, she hasn’t let…

UCLA researchers have identified the regulatory network that controls the transformation of neuronal progenitor cells into movement nerve cells in chicken and mouse embryos. The study adds insight into the development and functioning of spinal movement nerve cells. It also may contribute to the production of stem cell-derived movement nerve…

Neuraltus Pharmaceuticals signaled that it was moving toward the analysis stage of a Phase 2 clinical trial of its ALS therapy NP001. The signal was its announcement that it had completed the last patient visit in the study. Neuraltus conducted the trial (NCT02794857) to confirm the results of a…