Structure of TDP-43 Protein Clumps Identified for First Time

Structure of TDP-43 Protein Clumps Identified for First Time

A team of scientists in the U.K. and Japan has determined the structure of aggregated TDP-43, the protein whose abnormal clumps are characteristic of amyotrophic lateral sclerosis (ALS). Their work, reportedly the first to reveal the molecular structure of aggregated TDP-43, identified a “double-spiral fold” of the protein in patients’ brain…

Technology Blazes a Trail Out of the Wilderness

“Keep off the path, Beware of the gate, Watch out for signs that say ‘hidden driveways.’ Don’t let the chlorine in your eyes, Blind you to the awful surprise, That’s waitin’ for you at, The bottom of the bottomless, Blue, blue, blue pool, You’re livin’ in your own private Idaho.”…

We in the Rare Disease Community Can Learn From Each Other

ALS is a life-changing diagnosis, and like many newly diagnosed patients, I was disappointed and frustrated at what felt like a standstill in medical progress against the disease. “If they can send humans into space, why can’t they cure ALS?” I’d lament, Now, 11 years later and with still…

Cytokinetics’ Grants Support Patient Advocacy Groups

Cytokinetics has awarded a total of $100,000 to five nonprofit patient advocacy organizations to support efforts to enhance communications, increase disease awareness, and boost community engagement. Recipients of the fourth annual Communications Fellowship Grant Program include the Les Turner ALS Foundation, the Northern Ohio Chapter of The…

WVE-004 Trial in Patients With C9orf72 Mutations Gains Funding

Wave Life Sciences has gained additional financial support for an ongoing “basket” Phase 1b/2a clinical trial evaluating its experimental therapy, WVE-004, in people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene. So-called basket trials, commonly used in the field of cancer, test…

Eikonoklastes Acquires License to Potential ALS Gene Therapy

Eikonoklastes Therapeutics has added a candidate gene therapy for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases to its pipeline. The announcement follows the completion of a licensing agreement with the University of California San Diego. Brian Head, PhD, a professor with the university’s department of anesthesiology, and…

The Secret to Finding Balance as a Caregiver

“Kristin, I need my eyes wiped again,” my husband, Todd, calls from his office. I had just put drops in his eyes 20 minutes ago, hoping to address the incessant itchiness he was experiencing, and now we are back at it again. I get up from my desk, grab a…