Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
The European Medicines Agency (EMA) has granted orphan drug designation to Seelos Therapeutics‘ investigational therapy SLS-005 for the treatment of amyotrophic lateral sclerosis (ALS), the company announced in a press release. Orphan drug status in Europe is given to medicines with the potential to be safe and…
On one of my recent daily walks, I listened to an “Office Ladies” podcast in which Jenna Fischer shared her ongoing struggle with anxiety. She used the analogy of a backpack to describe the burden she lives with. Some days it weighs her down, while other days she…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…
AB Science will suspend recruitment and treatment in all studies involving masitinib, a tyrosine kinase inhibitor being investigated for amyotrophic lateral sclerosis (ALS) and several other conditions. The voluntary suspension follows the identification of a potential risk of ischemic heart disease — a condition of recurring chest…
Last week, I went to the doctor to get a problem with my ear checked out. The medical assistant went through a list of screening questions. The last was, “Are you depressed?” Tears welled up in my eyes. “Yeah, I’m depressed. My husband is terminally ill.” “I’m sorry,” she said.
We had a health scare a couple weeks ago, and I had to take our 8-year-old goldendoodle to the vet. Earlier in the week, Comet didn’t have his usual zip. Instead of eagerly greeting visitors, he stayed on his doggy bed. By Thursday evening of that week, Comet began…
The U.K. research organization LifeArc will match £500,000 (about $704,000) of the money raised by the director of the Leeds Rhinos’ rugby club in a series of marathons he ran to support research into amyotrophic lateral sclerosis (ALS) and other motor neuron diseases. The match for this part…
“You shoot me glances and they’re so hard to read, I misconstrue what you mean. … Are you just playing, making passes? Well, my heart could use some glasses. … Do I read you correctly, I need you directly,…
Two years after my husband, Todd, was diagnosed with ALS, I read an article that gave me hope. An Israeli rabbi who had ALS saw improvement with NurOwn, an experimental stem cell treatment by BrainStorm Cell Therapeutics. The man went from being in a wheelchair to walking. I…
