BrainStorm Cell Therapeutics met with U.S. Food and Drug Administration (FDA) officials regarding its plans for a semi-automatic manufacturing process for NurOwn, the company’s investigational cell-based therapy for amyotrophic lateral sclerosis (ALS). Discussion at the meeting — formally called a type C meeting — covered issues that…
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
Pridopidine is an investigational sigma-1 receptor agonist for the treatment of amyotrophic lateral sclerosis (ALS). Teva Pharmaceuticals was originally developing the treatment — which exhibits potential neuroprotective effects with a favorable safety profile — but Prilenia Therapeutics has since acquired the rights to further develop it. How…
I’m often asked by others in the ALS community for tips and insight from my past 10 years of living with ALS. While I am grateful for both making it this far while maintaining most of my physical abilities, I am by no means an expert. ALS symptoms differ…
Note: This story was updated Feb. 9, 2021, to clarify details surrounding the grant Chronic Care Collaborative received from Adira. Greg Smiley’s world changed abruptly in 2013. He was racing down a mountain road on an outback cycling trip in South Africa, when he hit an obstruction in the road…
If the sky above you, Grows dark and full of clouds, And that old north wind begins to blow, Keep your head together, And call my name out loud, Soon you’ll hear me knocking at your door. ……
It’s hard to find guides for the grief that accompanies a long, progressive terminal illness. Loss is often talked about as an event that happened in the past. It’s difficult to process grief when you know you haven’t reached the bottom yet, when the losses keep coming. In Brené Brown’s…
“Oh yes, I am wise, but it’s wisdom born of pain. — “I Am Woman,” Helen Reddy “Gain from pain” is not a new concept. The notion can be traced back several millennia to “The Ethics of the…
People with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases (MNDs), as well as their caregivers and healthcare professionals, are being recruited across the U.K. to provide input for the development of a web-based tool to help patients decide if they want a feeding tube. Patients are being…
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…
