People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
Scientist Manish Arora, PhD, received an $8 million Outstanding Investigator Award from the National Institute of Environmental Health Sciences (NIEHS) to develop models to predict and possibly prevent diseases such as amyotrophic lateral sclerosis (ALS). Arora and his team developed the Biodynamic Interface, which proposes an interplay…
Researchers at California’s Cedars-Sinai Medical Center have re-created the blood-brain barrier, a vital component of the central nervous system, using Organ-Chip technology by Emulate. This advances the possibility of patient-specific treatments for neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS). Blood-brain barrier defect has been linked to…
Low levels of membralin protein in the brain cells that support and protect neurons lead to the accumulation of the neurotransmitter glutamate to levels that induce the death of motor neurons in amyotrophic lateral sclerosis (ALS), a mouse study shows. A gene therapy that rescued the levels of…
ALS and efficient are two words not often used in the same sentence. But to me, they are a perfect pairing. Mention “ALS” and it’s easy to think slow, clumsy, and low energy, while “efficient” can describe quick, streamlined, and well-organized. I’ve combined the two into what I call…
News
AB Science’s New Masitinib Study Validated by EMA, With Expected Start Later This Year, Company Says
AB Science has optimized the design of a Phase 3 confirmatory study for mastinib, its oral treatment for amyotrophic lateral sclerosis (ALS), to avoid any possibility of bias. The optimized protocol for the study (NCT03127267), not yet recruiting, was validated by the European Medicines Agency (EMA). Although AB…
In the summer of 2014, the ALS Ice Bucket Challenge took social media by storm. Aimed primarily at raising awareness of the disease, the effort resulted in marked increases in spending on amyotrophic lateral sclerosis (ALS) research, an independent study shows. According to the report by…
MediciNova has launched the Phase 2b/3 clinical trial that will explore the potential of ibudilast (MN-166) in patients with amyotrophic lateral sclerosis (ALS). Pending the success of the trial, the collected data will support the submission of a new drug application and the approval of ibudilast…
As part of a series of profiles of people doing great things for their communities, the Obama Foundation has released a video spotlighting the efforts of a patient and his wife who want to reimagine the fight against amyotrophic lateral sclerosis (ALS). The Chicago couple, Brian Wallach…
A 1992 episode of the hit television series “Seinfeld” is a “life-imitating-art” story titled “The Pitch.” The character of George Costanza (with brief help from Jerry Seinfeld) attempts to explain the concept for a new show to fictional NBC executive Russell Dalrymple: George: I think I can sum…
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