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  • Groundbreaking ALS Voice of the Patient Report Submitted to FDA

    Posted by Dagmar on December 9, 2019 at 6:00 am

    The ALS Association recently submitted a report to the FDA documenting the real impact ALS has on people living with the disease and their caregivers in order to inform the development of treatments. The information in the report draws from a survey of people living with ALS.

    Key takeaways of the ALS Voice of the Patient Report:

    • Individuals with ALS and caregivers reported significant symptoms of fatigue and weakness, followed by speech problems, shortness of breath, difficulty sleeping, and pain.
    • These progressive symptoms result in a loss of independence and led to less time spent at work or school, socializing, or traveling.
    • People with ALS do not report high satisfaction with currently available treatments and reported that such treatments “somewhat help” with managing ALS and everyday impacts of disease. Breathing assistance devices were reported to be very inconvenient and burdensome, but necessary.
    • With current treatments mainly focused on supportive care, people with ALS would like treatment options that stop disease progression, improve muscle weakness, and assist with breathing or respiratory function.
    • The majority of people with ALS reported having fears about the future. The fears most frequently reported were related to dying, “leaving family too soon,” followed by “dying from respiratory failure.” The next most reported fear was “spending the family’s savings on medical care.”

    Read more here:

    Dagmar says: I agree with points 3 & 4, that people with ALS are not satisfied with current treatments and want more focus on treatment options that stop disease progression.

    What do you think about the results of this survey?

    john-russell replied 4 years, 2 months ago 3 Members · 3 Replies
  • 3 Replies
  • bill1949

    December 10, 2019 at 11:42 am

    Pretty obvious to all of us with ALS. Groundbreaking? I’m all for our voices to be heard though. I’m surprised that there wasn’t a high concern noted at the extremely slow pace of trials and disjointed treatment development process.

  • erin

    December 10, 2019 at 7:06 pm

    Look, I hate to sound pessimistic, but aren’t these results obvious? Who is the audience for this study? What was groundbreaking , the fact that actual pALS were used? Fears about the future, specifically dying too soon for a 100% fatal disease also seems logical .

    I certainly hope the FDA takes these voices and pours efforts behind approving real drug treatment that offers real hope.

    caregiver for pALS

  • Dagmar

    December 10, 2019 at 7:15 pm

    I agree with you Erin… points 1,2,3 & 5 are actually “duh” moments.

    What burns me is that in 2019 we still “need” a survey to verify 1 & 2.


  • john-russell

    December 12, 2019 at 8:16 pm

    I’m dumb founded. FDA didn’t really understand that creeping paralysis followed by suffocation and death is a problematic and upsetting diagnosis. Also unaware that anything to even slow progression much less stop it or even gain back loss would be appreciated by pALS?y

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