Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

Canadian authorities have approved the sale of Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS). The therapy will be marketed by Mitsubishi Tanabe Pharma Canada (MTP-CA), a unit of Mitsubishi Tanabe Pharma America. MTP-CA was established earlier this year to distribute Radicava and other medicines for difficult-to-treat…

Personalized assessment and intervention with a specific communication strategy are key to improving communication in patients with amyotrophic lateral sclerosis (ALS). Dysarthria, the term for a group of speech disorders, affects 95% of patients with ALS. Augmentative and alternative communication (AAC), which includes aided and unaided approaches such as communication books…

Psychological stress does not appear to play a part in the development of amyotrophic lateral sclerosis (ALS), with patients showing similar levels of prior stressful events, occupational stress, and anxiety as a control group, as well as higher resilience, a study shows. The study, “Is psychological stress…

Investigational compound EPI-589 was found to be safe and well-tolerated, improved biomarkers of neuroinflammation, and showed signs of slowing disease progression in amyotrophic lateral sclerosis (ALS) patients, according to a completed Phase 2a clinical trial. BioElectron Technology Corporation’s EPI-589, or (R)-troloxamide quinone, is a 250 mg immediate-release, film-coated…

The 2012 comedy “Ted” contained a joke made at the expense of the movie’s villain. The joke, as expressed by a teddy bear come to life, was: “From one man to another, I hope you get Lou Gehrig’s disease.” This alarmed some ALS patients and advocates, who…

Bulbar-onset amyotrophic lateral sclerosis (ALS) is the most common form of the disease among patients 80 and older at disease onset, a factor that seems to contribute to shorter survival for these patients compared with younger ALS patients, a retrospective study has found. The study, “Very late-onset amyotrophic…

For those of us living with ALS, every day can have a stressful event. The key, I believe, is to move through the event and, if possible, remember it with humor. I recently came across a quote from the movie “Slow West” that reminds me that…

FUS  — a protein mutated in some amyotrophic lateral sclerosis patients  — plays a key role in the mechanism that repairs oxidative damage in DNA molecules in motor neurons, a study found. The findings suggest a mechanism through which FUS defects cause amyotrophic lateral sclerosis (ALS), and add to the…

Medicines used for ovarian and breast cancer, called PARP inhibitors, might be repurposed to treat people with amyotrophic lateral sclerosis (ALS), a study in cells suggests. This suggestion came from researchers’ work with veliparib (ABT-888), an investigational PARP inhibitor, that found it effectively reduced TDP-43 aggregates — a hallmark of…