Amyotrophic lateral sclerosis (ALS) is a progressive neurological disease that affects motor neurons or nerve cells that control muscle movement. There is currently no cure for ALS but there are experimental treatments, including gene therapy. ALS and genetics In ALS, some genes are known to be modified or mutated, which…
UniQure is developing an experimental gene therapy for amyotrophic lateral sclerosis (ALS) using its proprietary, next-generation gene-silencing platform called miQure. ALS is a neurodegenerative disease characterized by the progressive loss of motor neurons — the nerve cells that control movement. ALS is progressive, meaning that symptoms worsen over…
SynCav1 is an experimental gene therapy for the treatment of neurological disorders such as ALS and Alzheimer’s disease. First developed at the University of California San Diego, SynCav1 has been licensed to CavoGene LifeSciences. How SynCav1 works In the brain, a protein called caveolin-1 is found…
*** Instant Karma’s gonna get you, Gonna knock you right on the head, You better get yourself together, Pretty soon you’re gonna be dead. — John Lennon *** One of the dictionary definitions of karma…
For more than four decades, comedian Jerry Lewis hosted the MDA Labor Day Telethon on behalf of the Muscular Dystrophy Association. That annual event helped the MDA become the nation’s largest non-government source of funding for neuromuscular disease research, with more than $1.4 billion disbursed since its establishment 68…
Mitsubishi Tanabe Pharma America (MTPA), in collaboration with the Massachusetts General Hospital (MGH), is conducting a study to identify biomarkers related to amyotrophic lateral sclerosis (ALS), which may be helpful to measure patient response to Radicava (edaravone). The study will be sponsored by MTPA, Radicava’s seller in…
“I think, therefore I am.” I have long been fascinated by that argument of philosopher Rene Descartes. Similarly, over a millennium earlier, St. Augustine wrote, “I make mistakes, therefore I am.” Both suggest that cognition — self-awareness in Descartes’ case, knowing right from wrong per St. Augustine —…
Higher levels of apathy — particularly its behavioral symptoms — in people with amyotrophic lateral sclerosis (ALS) are associated with a degree of greater burden for caregivers, according to a new study. The research, “The burden of apathy for caregivers of patients with amyotrophic lateral sclerosis,”…
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term…
“Youth is wasted on the young.” That quote, often attributed to playwright George Bernard Shaw among others, may on the surface appear paradoxical. However, to me its meaning is clear. When we are young and in our physical prime, both the slate of options for physical…
