Living with ALS certainly has its challenges, which lead many of us to rely on workarounds. These are creative, temporary solutions that solve an everyday problem; I first wrote about mine in “The ALS Workaround Dilemma.” But the key word here is “temporary,” because workarounds run the risk…
About a year ago, I wrote about how ALS patients may receive home healthcare on an uninterrupted basis, with expenses covered by Medicare. Since then, there has been both good news and bad news. The good news is that I recently “celebrated” my two-year anniversary of getting that coverage.
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…
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…
