Orphazyme Partnership to Run Through Extension of Phase 3 Arimoclomol Study

Orphazyme announced that it and Worldwide Clinical Trials will continue their collaboration through the long-term extension of a Phase 3 study into arimoclomol, a potential oral treatment for amyotrophic lateral sclerosis (ALS). ORARIALS-01 is a randomized, placebo-controlled and double-blind trial (NCT03491462) underway at 30 centers across North America…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Would it surprise you to learn that I check my cellphone zero times a day? All around me, people are texting, emailing, chatting, and checking social media. Me? Nada. My eyes are forward, watching the world. I’m embracing “the joy of missing out” — a state of mind caused by…

Amyotrophic lateral sclerosis (ALS) progression is associated with higher levels of specific lipids in the spinal cord and changes in their metabolism, a study in a rat model of the disease reports. These findings may represent a defense mechanism against oxidative damage, as well as a potential treatment route for…

A newly developed robotic neck brace may improve quality of life for people with amyotrophic lateral sclerosis (ALS) and allow researchers a more detailed assessment of head and neck movements, as well as disease progression, for people with this condition. The brace was described in a pilot study, titled…

People with rapid weight loss in the earlier stages of amyotophic lateral sclerosis (ALS) — before invasive ventilation is needed to support breathing — are more likely to have a poorer prognosis in its later stages than those who don’t, a study reports. The study, “Body weight variation predicts…

“What? But there’s a guarantee on your website,” I told the customer service representative for the herbal company. I had requested a refund of a digestive supplement because it didn’t work for me. He repeated his scripted line: “That product is nonreturnable because it’s consumable.” “Wow!” I couldn’t believe my…

A $3.1 million grant from the National Institute on Aging (NIA) is intended to help two scientists at Northwestern University find new treatments for amyotrophic lateral sclerosis (ALS). The grant was awarded to P. Hande Ozdinler, PhD, a professor of neurology at the university’s Feinberg…

The spark of inspiration for column topics sometimes comes to me from surprising sources. Last Saturday, with my submission deadline looming, I had nothing … nada … bupkis. Fearing I would to have to forgo my weekly passion, I passed the time reading, with some music playing in…

Pharmaceutical company MSD and the Francis Crick Institute are partnering to try to learn more about what causes motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS), so that treatments may be developed. Funded by MSD and the Medical Research Council, the project…