Top Tweets for #ALSFRS

almost 4 years ago

"We KNOW #NurOwnWorks" ~@GoettlShelly (11.9.22) ▪️May 2017 #ALS thumb onset Roberto's hand paralyzed ▪️Aug 30 #NurOwn EAP dose Regained function in his fingers & strength in arm. Now he can operate his own wheelchair! ZERO pts on #ALSFRS but "clinically meaningful" for QOL!

aVoice4ALS's tweet photo. "We KNOW #NurOwnWorks"
~@GoettlShelly (11.9.22)

▪️May 2017 #ALS thumb onset
Roberto's hand paralyzed

▪️Aug 30 #NurOwn EAP dose
Regained function in his fingers & strength in arm. Now he can operate his own wheelchair!

ZERO pts on #ALSFRS but "clinically meaningful" for QOL! https://t.co/YzNX6hEh1T

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Nicole Cimbura @nicolecimbura

over 4 years ago

What was your child doing when he was a teen? Playing football? Video games? Fishing trips? Skiing? Or emptying catheters for a parent who lost 1 pt on the #ALSFRS? @US_FDA please #TriageALS & act with same urgency that #ALS acts on life. @FDACDERDirector #everypointmatters

over 4 years ago

Dear @US_FDA when you delayed @AmylyxPharma decision did you think of kids missing out on activities b/c of caregiving duties?Loss of pts in the dressing & hygiene category of the ALSFRS-R scale equates to loss of independence.#everypointmatters @HealthCanadaNOC understands it!

nicolecimbura's tweet photo. Dear @US_FDA when you delayed @AmylyxPharma decision did you think of kids missing out on activities b/c of caregiving duties?Loss of pts in the dressing & hygiene category of the ALSFRS-R scale equates to loss of independence.#everypointmatters @HealthCanadaNOC understands it! https://t.co/pbPMaUmN08

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Nicole Cimbura @nicolecimbura

over 4 years ago

Dear @US_FDA with the delay of the @AmylyxPharma decision did you think of #everpointmatters on the #ALSFRS-R?Loss of speech w/ non vocal comm(1pt) to loss of useful speech(0pt) landed Mike in the hospital. It was over Thanksgiving break so we had to move traditions there, uurgh!

nicolecimbura's tweet photo. Dear @US_FDA with the delay of the @AmylyxPharma decision did you think of #everpointmatters on the #ALSFRS-R?Loss of speech w/ non vocal comm(1pt) to loss of useful speech(0pt) landed Mike in the hospital. It was over Thanksgiving break so we had to move traditions there, uurgh! https://t.co/OCbkmbSMMu

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Farmstrong @farmstronginfo

over 4 years ago

#everypointmatters A point on the #ALSFRS scale is the difference between walking and not walking. Rolling yourself in bed or waking a loved one. Breathing on your own or with a machine. #dyingWaiting @RepScottPerry @PeteAguilar @RepRaulRuizMD @Team_Thriving @jackiesmom12

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Farmstrong @farmstronginfo

over 4 years ago

Unless you have #ALS, you don't understand that a point on the #ALSFRS scale means everything. It also opens the door for future function gain (points). Few know this but @US_FDA should bc we tell them hundreds of times daily & in every meeting w/ them.

Nicole Cimbura @nicolecimbura

over 4 years ago

Dear @FDA when delaying the @AmylyxPharma decision by 3 months did you consider that #everypointmatters? It can equate to telling my kids "I love you" in my voice instead of via machine.@klink52 @ThurmanTough @SarahNauser @JamieRoseBerryy what does 1 pt mean to you?@RepJasonCrow

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over 4 years ago

A hippo, dog & squirrel were each given 2 hunger tablets, then observed to see which could eat most. Conclusion: the hunger tablets worked best on the hippo, followed by dog, then squirrel. Have I told u the one about the #ALSFRS & heterogeneity of #ALS? 🦛 @US_FDA @DrCaliff_FDA

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over 4 years ago

...and @califf001 @FDACBER If I were to lose the ability to stand from a crouched position, the #ALSFRS-R would not care. It doesn't fit under any question. What happens if I regain the ability to stand from a crouched position? Nothing with the ALSFRS, but I'd be one happy dad.

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axeALSFoundation @stevens_nation

almost 5 years ago

2+ yrs ago, Eric was losing 2 pts/mo on #ALSFRS scale Thx to #NurOwn, he can still push his daughter's stroller, read her a book, bathe, feed her & play with her. Eric is #LivingProof that #NurOwnWorks @BrainstormCell #FileTheBLA so Eric can keep making memories #GirlDad #ALS

over 5 years ago

Eric can still get down on the floor to play w/ our 5 mo old daughter. He gets up by himself using the couch for help. 2 years ago, doctors said he probably wouldn’t be here. Well, not only is he here—he’s singing, laughing & playing w/ our little girl. #LivingProof #NurOwnWorks

stevens_nation's tweet photo. Eric can still get down on the floor to play w/ our 5 mo old daughter. He gets up by himself using the couch for help. 2 years ago, doctors said he probably wouldn’t be here. Well, not only is he here—he’s singing, laughing & playing w/ our little girl. #LivingProof #NurOwnWorks https://t.co/6v6eflB1qK

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almost 5 years ago

In the @CleneNano CNM-Au8 Phase 2 study in #ALS, note the difference between bulbar onset vs limb onset. Good evidence that drug has significant impact on limb onset. #MND #ALSFRS

aVoice4ALS's tweet photo. In the @CleneNano CNM-Au8 Phase 2 study in #ALS, note the difference between bulbar onset vs limb onset. Good evidence that drug has significant impact on limb onset.

#MND #ALSFRS https://t.co/hyupwhImEQ

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almost 5 years ago

Jeopardy Contestant: I'll take #ALSFRS-R for $800, Alex! Alex Trebek: Objects that can decide an #ALS drugs efficacy. Jeopardy Contestant: What are Pillows & Handrails! Alex Trebek: Correct! The @US_FDA uses a 30 year old questionnaire to decide if an ALS drug works! #NurOwn

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almost 5 years ago

Do you use the hand rails when using the stairs? This is a 3 points question on the #ALSFRS-R. @US_FDA @FDACBER #FDA uses the ALSFRS-R questionnaire for #ALS drug approval, but has no interest in the patient reported outcomes reported like improved function. #NurOwn #NurOwnWorks

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Kylan Morris @KylanMorris1

about 5 years ago

1 pt improvement in SWALLOWING on #ALSFRS-R & HUGE improvement in QOL. #NurOwn improved function & Sandy just proved it! #LivingProof #NurOwnWorks @FDACBER @DrWoodcockFDA @WHCOS @SecBecerra @HHS_ASH @RepAnnaEshoo @rosadelauro @RepSchakowsky @SenFeinstein @SenAlexPadilla

about 5 years ago

Before getting #NurOwn in EAP, mama could barely eat/swallow soft foods without difficulty. This weekend, our clients gifted us delicious sourdough bread. Mom ate everything you see on this plate - crispy, delicious crust & all! Thank you @BrainStormCell and @Cylebo 1/2

KylanMorris1's tweet photo. Before getting #NurOwn in EAP, mama could barely eat/swallow soft foods without difficulty. This weekend, our clients gifted us delicious sourdough bread. Mom ate everything you see on this plate - crispy, delicious crust & all! Thank you @BrainStormCell and @Cylebo 1/2 https://t.co/TPNR4A8g1C

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about 5 years ago

Imagine if scales didn't exist and you had to use the ALSFRS to guess how much you weighed? You'll have to choose from Light, Average, & Heavy. Imagine if a drugs efficacy was decided this way? Welcome to #ALS. #ALSFRS @FDACDERDirector @FDACommissioner RepAnnaEshoo @rosadelauro

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over 5 years ago

PI Neurologists said they couldn't testify to "reasonable degree of medical certainty" re a dx based solely on subjective #ALSFRS score. If it wouldn't withstand cross exam, why use it deny efficacy of $50M #ALS trials & block access to drugs for 450,000 lives? #ALSHearings

aVoice4ALS's tweet photo. PI Neurologists said they couldn't testify to "reasonable degree of medical certainty" re a dx based solely on subjective #ALSFRS score. If it wouldn't withstand cross exam, why use it deny efficacy of $50M #ALS trials & block access to drugs for 450,000 lives?

#ALSHearings https://t.co/hSyiVPtHk6

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over 5 years ago

@ScottFightsALS So glad it a a good day! Great evidence of flaw in measuring tool ... but no where does this triumph get reflected in the #ALSFRS-R score. Flawed tool = flawed data = flawed statistical conclusions re #ALS efficacy & "substantial evidence"

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