To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
“Can you believe our little Fafa finished ninth grade?” I asked my husband, Todd, as I flushed his feeding tube. He laughed, and we took a trip down memory lane. Our daughter, Sara, gave herself the nickname. Before she was even 2, she said something like, “Fafa wants that.”…
“Welcome to my nightmare, whoa, “Welcome to my breakdown, “I hope I didn’t scare you …” If there were a “Welcome Wagon” function in the ALS community — whereby incumbent residents indoctrinated new arrivals — surely the introduction would resemble those lyrics from Alice Cooper. Grim statistics and personal…
Lately, I’ve noticed a strange trend happening in several online ALS communities I belong to. I’ve seen an increase in posts from people who haven’t yet been diagnosed with ALS asking members of the group to essentially offer medical opinions. A typical post begins with a list of physical symptoms,…
Amylyx Pharmaceuticals is seeking approval, in Canada, of its lead candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). This regulatory submission “represents a significant milestone in our efforts to develop a new treatment option for people living with ALS who have no time to wait,” Joshua Cohen, co-founder, co-CEO,…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
On occasion, I’ll page through the photo books my husband, Todd, made for me as Christmas gifts. I feel a sense of gratitude as I look back on all we’ve been able to do since his ALS diagnosis. Family game nights. Outings to sporting events. Family movie nights. Several…
“No one knows what it’s like, “To be the bad man, “To be the sad man, “Behind blue eyes.” – The Who Last week, it was time for the annual agency performance audit of my aides. Actually, owing to COVID-19, the one for 2020 hadn’t taken place. Consequently, the…
The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Spinogenix’s investigational oral molecule SPG302 for treating amyotrophic lateral sclerosis (ALS). Orphan drug status is granted by the FDA to encourage the development of therapies for conditions that affect fewer than 200,000 people in the…
