Stumbling Upon the Sublime in a Minefield of the Ridiculous
“Well I don’t know how to tell the weight of the sun, … How the home computer has me on the run, … Of all the Nobel prizes that I’ve never won, … Please be upstanding for the Mayor of…
Stumbling Upon the Sublime in a Minefield of the Ridiculous
NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…
Two ALS Staging Systems May Be Useful Assessment Tools in Clinical Trials
Two amyotrophic lateral sclerosis (ALS) staging systems — the King’s and the Milano-Torino’s, known as MiToS — may effectively assess changes in patients’ clinical progression and treatment responses, a study suggests. The data also supported the previously reported complementary nature of these two systems, showing that the…
Zooming Past My Previous Predictions
During these final weeks of 2020, I’ve been reflecting on the various ways this world health crisis has affected the ALS community. One evolving activity that has held my interest ever since I included it in my post-pandemic predictions in a column last April is online ALS support groups.
Polio-related Research Into Brain Motor Network Shows Potential ALS Marker
New research into the motor networks of polio survivors shows the brain may “rewire” itself in neurological diseases, leading to a reorganization that also may serve as a potential biomarker of amyotrophic lateral sclerosis (ALS) and other motor neuron disorders, a study reports. Published in…
First Set of Guidelines for ALS Care, Management in Canada Published
A first set of Canadian guidelines for the care of people with amyotrophic lateral sclerosis (ALS) have been published, emphasizing the need for a holistic and patient-centered approach, with attention to emotional well-being. The guidelines, reported to include comprehensive and easy-to-reference best practice…
Survey Finds COVID-19 Disrupted Care, Well-being of Rare Disease Patients in Europe
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
Giving Thanks With 2020 Vision
“I am a rock, I am an island, And a rock feels no pain.” In 2020, there have been none of Paul Simon’s “rocks” to be found in the social circles that I inhabit. Everyone that I…
BREN-02, Potential Restorative ALS Therapy, Named Orphan Drug by FDA
BREN-02, a lab-made form of the human protein engrailed 1 (EN1), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA) as a potential treatment of amyotrophic lateral sclerosis…
Dosing Begins in Phase 2 Study of Pegcetacoplan in ALS Patients
A first patient has been dosed in the Phase 2 trial investigating pegcetacoplan (APL-2), Apellis Pharmaceuticals‘ candidate therapy for amyotrophic lateral sclerosis (ALS), the company and the Swedish Orphan Biovitrum (Sobi) announced. The potentially pivotal trial, called MERIDIAN (NCT04579666), is currently enrolling patients…
Recent Posts
- Access to investigational ALS therapy MN‑166 expanded further
- AUT00201 boosts muscle strength, coordination in ALS mouse model
- People with ALS should be able to proactively get feeding tubes
- Zhimeng cleared to launch study of ALS therapy CB03-154 in China
- How I’m now dancing to the beat of my own ALS