The former malaria therapy pyrimethamine reduces levels of an ALS-linked protein in the spinal fluid of patients whose disease stems from gene mutations, a Phase 1/2 clinical trial shows. The protein is SOD1. The mutations connected with amyotrophic lateral sclerosis are in the SOD1 gene that controls the protein. The…
Mallinckrodt has launched the Phase 2b PENNANT trial (NCT03068754) to investigate the safety and effectiveness of H.P. Acthar Gel (repository corticotropin injection) as a treatment for amyotrophic lateral sclerosis (ALS). The H.P. Acthar Gel is an experimental injectable drug that the U.S. Food and Drug Administration (FDA) has granted fast…
The U.S. Food and Drug Administration has expanded its approval of Dysport (abobotulinumtoxinA) as a treatment for spasticity in adults from not just the upper limbs but to the lower limbs as well, according to its maker, Ipsen Biopharmaceuticals. Spasticity can affect those with amyotrophic lateral sclerosis (ALS), multiple sclerosis, cerebral…
Stem cell transplantation in patients with amyotrophic lateral sclerosis (ALS) has the potential to be “an important alternative strategy” in treating the disease, a new study suggests. In recent decades, many advances in identifying and understanding the underlying mechanisms of ALS have been made. But those advances have not resulted in…
A new study shows that patients with amyotrophic lateral sclerosis (ALS) experience decreased oxidative metabolism during exercise, and that differences among individuals in exercise capacity matches ALS clinical heterogeneity. The study, titled, “Inefficient skeletal muscle oxidative function…
AveXis and REGENXBIO have signed a new licensing agreement to develop therapies for a particular form of ALS and for Rett syndrome. They already have an agreement that covers spinal muscular atrophy therapies. The form of amyotrophic lateral sclerosis that the new agreement covers is the one caused by mutations…
ALS patients compensate for neuron loss in some brain regions by stepping up activity in other regions, according to a study. The finding suggests that analyzing patients’ brain networks could help doctors determine the state of their disease and monitor its progression. The study, “Functional reorganization during cognitive function…
People dying of terminal diseases like amyotrophic lateral sclerosis (ALS) and inmates on death row have something in common: their outlooks are more positive than might be expected. A recent study examining attitudes on impending death, “Dying is Unexpectedly Positive,” appeared in the journal Psychological Science. Death is an important part…
Flex Pharma, which develops therapies to reduce the cramps and shaking that are hallmarks of ALS and other neurological diseases, has a new CEO. Dr. William McVicar, head of the company’s research and development arm, will assume the interim chief executive’s post on July 3. He replaces Dr. Christoph Westphal, who…
American Airlines has raised more than $800,000 at its second annual American Airlines Charity Golf Tournament to support the research initiative Answer ALS. The tournament and a fund-raising auction took place in American’s home base of Dallas, May 24 and 25. “We are proud to partner with Answer ALS…
