Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
ALS has compromised the breathing of my husband, Todd, to the point where his positioning — unless he is using the Astral noninvasive ventilator — can mean the difference between being able to breathe or not. We only realized how bad it was a couple of weeks ago when we…
Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…
Every few months, I stop and check in on what’s working in my life and what’s not. It’s a personal wellness ritual that’s served me well in the years I’ve been living with ALS. Today, I’m evaluating how well I’ve been following my intention for 2022 to “simply…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
The European Medicines Agency (EMA) has agreed to review a marketing authorization application (MAA) from Amylyx Pharmaceuticals requesting the approval of AMX0035 — a combination of two compounds — for the treatment of people with amyotrophic lateral sclerosis (ALS). The validation comes nearly two months after Amylyx filed the…
I set my husband, Todd, up on his computer with his HeadMouse and sip-and-puff clicker. ALS has compromised his breathing, so I put on his noninvasive ventilator. I call my mom, who lives next door. She will keep her mobile phone in her pocket. If Todd needs anything, he…
Health Canada has authorized a new drug submission for masitinib — AB Science’s add-on therapy for amyotrophic lateral sclerosis (ALS) — under the notice of compliance with conditions policy (NOC/c), designed to get new medications to patients more quickly. If granted, this would allow conditional marketing of…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
“Will you still need me, will you still feed me, When I’m sixty-four” Paul McCartney wrote the song “When I’m Sixty-Four” four decades before reaching that age himself. McCartney admitted that the number was chosen arbitrarily. He later quipped during an interview that 65 would have been a…
