Published today in ALS News Today
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Apic Bio‘s APB-102, an investigational gene therapy aiming to treat familial amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene.
For the backstory on this thread, please see the original Familial ALS posting. Perhaps a moderator can combine these two?
We have received the gentetic testing results from my husbands brother, who had ALS-like neuromuscular degeneration combined with a family tree with many ALS and...
Hello everybody, I have a question that I felt like couldn't be answered in the Stickies.
For about 3 weeks I have had issues where I am walking, and if I do a quick turn on one leg, I stumble a bit. I have gotten out of bed at night to have my legs shake uncontrollably, or I stumble around the...
One of my daughter’s first child had Cystic Fibrosis, which is a genetically passed condition. So they could have additional children with NO risk of CF they elected to have In vitro fertilization for the next children. Embryos were created and tested and only CF free embryos were frozen. IT...
long story short. my grandfathers side of the family has familial als. My grandfathers brother got the disease and died within two years. there mother had als and there grandmother also had als. although my grandfather did not get als. neither did his other 3 sisters or other brother get the...
Hi everyone - I am seeing my primary care doctor tomorrow but wanted to get your opinion on my situation seeing as you have so much experience in this area.
My uncle passed away in Spring 2017 from ALS (he was late 60s) and my dad was just diagnosed with ALS (he is 74). I'm considering getting...
Thank you for adding me/us to the forum. In brief, here is our story: My husband lost his mother to ALS when she was 59 years old. She had one aunt, one uncle, and two first cousins who also passed from ALS. There were a couple other family members who may or may not have had early signs, but...
I am new here, so please bear with me if I break any rules, and feel free to delete this thread.
My grandfather passsed away aged ~65 from ALS after having been ill for 4 years. His brother still suffers from ALS, but he was diagnosed much earlier, almost 20 years ago. His illness moves...
I was recently diagnosed with Onset Bulbar ALS. The question came up about history and I told the Neuro that my grandfather, a cousin and 2 of my grandfathers nieces had it. My understanding is that to connect it as familial my grandfather would have had to had a genetics test for them to be...