Throughout February, many patient columnists like me who write for Bionews, the parent company of this website, are writing columns recognizing Rare Disease Month, which culminates in Rare Disease Day on Feb. 28. Not only are we encouraging our readers to reach across patient community lines to learn…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
A U.S. Food and Drug Administration (FDA) advisory committee has scheduled a virtual meeting with Amylyx Pharmaceuticals to review its application for the approval of AMX0035 to treat amyotrophic lateral sclerosis (ALS), the company announced. The online meeting to discuss data supporting the approval request is set for March 30.
Our daughter, Sara, is working on several pieces for an upcoming choir festival. One selection is the song “My New Philosophy” from the musical “You’re a Good Man, Charlie Brown.” The other evening, as our family gathered for dinner, Sara sang the lyrics in which the character Sally tries…
Since February is known as the month of love, I’d like to shower some love on all the ALS caregivers around the world. Add a special shoutout to family caregivers — the spouses, partners, family members, friends, and even neighbors who care for someone with ALS. It’s a challenging responsibility…
The Cullen Education and Research Fund (CERF), a private philanthropy based in London, has awarded more than $800,000 to support two U.S. research projects that are working to improve life for people with amyotrophic lateral sclerosis (ALS). The CERF Medical Engineering Prize, worth €500,000 (about $570,000), was awarded to…
I’ve been worried about leaving my husband, Todd, who has ALS, alone after his close call with his breathing last week, but he said he would be fine while I ran to town. I needed to pick the kids up from school and take our daughter to a dentist…
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
The Canadian Neuromuscular Disease Registry (CNDR) and Amylyx Pharmaceuticals are collaborating on an initiative that could produce the first real-world evidence on AMX0035, Amylyx’s investigational therapy to slow functional decline in amyotrophic lateral sclerosis (ALS) patients. The two, in partnership with neuromuscular centers across Canada, will collect…
More than half of the people with amyotrophic lateral sclerosis (ALS) responding to a survey were dissatisfied with their quality of life and pessimistic about their future. The U.S.-based survey was conducted by ALS News Today. Nevertheless, the vast majority of respondents reported to be satisfied with their…
