car128

@DrMakaryFDA But what about those of us suffering from 100% fatal ALS. You have had our citizens petition for over 170 days. We need #NurOwn to have accelerated approval now!! Why do we continue to suffer while you do nothing.

Under President Trump’s leadership, the FDA: ✅ Just hit a monthly record cell & gene Tx approvals ✅ Hitting ALL drug target date goals ✅ Eliminating animal testing requirements ✅ Reducing decision times (1 yr to months) ✅ Onboarding 1,000 new scientists ✅ Accelerating cures, cutting red tape ✅ Allowing real world evidence ✅ Making decision letters public ✅ Incentivizing domestic manufacturing ✅ Cutting biosimilar develop costs in half ✅ Moving more meds to “over the counter” ✅ Telling the truth on HRT for women ✅ Removing all 9 artificial food dyes ✅ Re-writing the broken food pyramid ✅ Closing the GRAS loophole ✅ Modernizing infant formula ✅ New leadership, a new era of innovation The FDA is strong 💪💪 and will continue to be 💪💪

.@FDACBER @VPrasadMDMPH, you brought hope with your words, but did you mean what you said? 127 DAYS you've had our Nurown Citizens petition=SILENCE ALS=100% FATAL since 1869 @SecKennedy @DrMakaryFDA, Every day of delay=more HORRIFIC deaths WITHOUT HOPE. No words can convey how HORRIFIC the suffering is while we wait on the FDA GRANT ACCELERATED APPROVAL for NurOwn NOW. Our Citizens' Petition, submitted on July 3rd and now ignored for 127 days, demands it. This autologous stem cell therapy—our own cells engineered to flood our brains with life-saving neurotrophins—is ALS's FIRST BREAKTHROUGH in 156 years of hell. The Phase 3b trial was cleared in May 2025, and the data from the 2022 ADCOMM screams for approval. Irrefutable Evidence Biomarkers PROVE It Works: Reduced Neuroinflammation: Significant decrease in MCP-1 cytokines (p<0.05). Reduced Neurodegeneration: Significant drop in Neurofilament Light Chain (NfL) (p<0.05). Increased Neuroprotection: Massive surge in neuroprotective growth factors like GDNF/VEGF (p≤0.001). A total of 23 signals confirm target engagement. Real-World Survival SHATTERS Norms: The Expanded Access Program (EAP, n=10) shows 100% 5-YEAR survival without tracheostomies, compared to only 20% in the natural history of the disease (p=0.006). The median tracheostomy-free survival is extended to 7 YEARS (a 2-3x extension). Progression was halted for up to 17 months, and 85% of participants saw a SLOWED decline (ALSFRS-R: 0.15 pts/mo vs. natural freefall). Navy veteran Matt Bellina GAINED 6 points on the ALSFRS-R scale after 7 doses. ALS Trials are Rigged Against Hope: Current trials suffer from small sample sizes (n=200), extreme patient heterogeneity, and the flawed ALSFRS-R questionnaire primary endpoint. NurOwn has shown success despite these barriers. The TOFERSEN Precedent: Tofersen was approved based on an NfL drop alone for just 2% of the ALS population. NurOwn has demonstrated broader, better survival benefits supported by extensive real-world evidence & ALSO HAD A STATISTICALLY SIGNIFICANT REDUCTION in Nfl. A conditional approval with a Phase 4 follow-up is the clear path forward. A Direct Appeal to FDA Leadership @MartyMakary: You have vowed to combine "gold standard science + common sense" to give rare disease warriors faster access. We are asking you to deliver on that promise for cell therapies. @RobertKennedyJr: Your crusade for stem cell therapies and your goal to end the FDA's "war" on them directly targets the crisis facing veterans and the underserved who are massacred by ALS. Thousands are dying during these delays. Fulfill your promise. Bureaucracy KILLS. Evidence SAVES. Read the petition: nurownworks.com/read-the-petit… Convene a meeting TODAY. Give us a fighting chance to LIVE. #BeyondSignsOfPromise #ApproveNurOwn #EndALS156YearsOfHopelessness @realDonaldTrump @POTUS @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy

ALS lives have never been a priority at the @FDACBER. I hoped that would change with @DrMakaryFDA & @VPrasadMDMPH, but there hasn't been anything more than the empty promises we have heard while we die. @MartyMakary, you have had our Nurown Citizens petition, with unprecedented gold standard survival data, for 124 DAYS = SILENCE, while #ALS ravages our bodies and thousands die without hope How many more families have to bury loved ones before the @US_FDA treats 100% FATAL #ALS with the same urgency & flexibility as terminal cancer treatments? 156 years of hopelessness continue for #ALS patients & our families.

.@FDACBER @VPrasadMDMPH @DrMakaryFDA, you brought hope with your words, but did you mean what you said? 138 DAYS you've had our Nurown Citizens petition=SILENCE ALS=100% FATAL since 1869 @SecKennedy, Every day of delay=more HORRIFIC deaths WITHOUT HOPE. No words can convey how HORRIFIC the suffering is while we wait on the FDA GRANT ACCELERATED APPROVAL for NurOwn NOW. Our Citizens' Petition, submitted on July 3rd and now ignored for 138 days, demands it. This autologous stem cell therapy—our own cells engineered to flood our brains with life-saving neurotrophins—is ALS's FIRST BREAKTHROUGH in 156 years of hell. The Phase 3b trial was cleared in May 2025, and the data from the 2022 ADCOMM screams for approval. Irrefutable Evidence Biomarkers PROVE It Works: Reduced Neuroinflammation: Significant decrease in MCP-1 cytokines (p<0.05). Reduced Neurodegeneration: Significant drop in Neurofilament Light Chain (NfL) (p<0.05). Increased Neuroprotection: Massive surge in neuroprotective growth factors like GDNF/VEGF (p≤0.001). A total of 23 signals confirm target engagement. Real-World Survival SHATTERS Norms: The Expanded Access Program (EAP, n=10) shows 100% 5-YEAR survival without tracheostomies, compared to only 20% in the natural history of the disease (p=0.006). The median tracheostomy-free survival is extended to 7 YEARS (a 2-3x extension). Progression was halted for up to 17 months, and 85% of participants saw a SLOWED decline (ALSFRS-R: 0.15 pts/mo vs. natural freefall). Navy veteran Matt Bellina GAINED 6 points on the ALSFRS-R scale after 7 doses. ALS Trials are Rigged Against Hope: Current trials suffer from small sample sizes (n=200), extreme patient heterogeneity, and the flawed ALSFRS-R questionnaire as the primary endpoint. NurOwn has shown success despite these barriers. The TOFERSEN Precedent: Tofersen was approved based on an NfL drop alone for just 2% of the ALS population. NurOwn has demonstrated broader, better survival benefits supported by extensive real-world evidence & ALSO HAD A STATISTICALLY SIGNIFICANT REDUCTION IN Nfl. Accelerated Approval with a Phase 4 follow-up is the clear path forward. A Direct Appeal to FDA Leadership @MartyMakary: You have vowed to combine "gold standard science + common sense" to give rare disease warriors faster access. We are asking you to deliver on that promise for cell therapies. @RobertKennedyJr: Your crusade for stem cell therapies and your goal to end the FDA's "war" on them directly targets the crisis facing veterans and the underserved who are massacred by ALS. Thousands are dying during these delays. Fulfill your promise. Bureaucracy KILLS. Evidence SAVES. Read the petition: nurownworks.com/read-the-petit… Convene a meeting TODAY. Give us a fighting chance to LIVE. #BeyondSignsOfPromise #ApproveNurOwn #EndALS156YearsOfHopelessness @realDonaldTrump @POTUS @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy

.@FDACBER @SecKennedy, It's 90 DAYS since our #NUROWN Citizens' Petition Survival data: #GoldStandard for FDA approvals @DrMakaryFDA said, 'gold standard science & common sense' guide this FDA. I don't understand the delay #NUROWN survival data UNPRECEDENTED in ALS history & UNAVAILABLE at ADCOMM Five-Year Survival: 100% in NurOwn EAP vs. 20% in ALS natural history. All EAP participants (n=10) achieved five-year survival without tracheostomies. Extended Tracheostomy-Free Survival (TFS): 7-year median tracheostomy-free survival (range from 5 to 8.5 years), far surpassing the 2.5- to 3-year median in ALS natural history data. Progression-Free Survival (PFS): When patients received NurOwn, they experienced PFS ranging from a few months up to 17 months. Every day of delay dooms more patients. The suffering is immeasurable. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-petit… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy zo

.@FDACBER @SecKennedy @DrMakaryFDA, It's 89 DAYS since our #NUROWN Citizens' Petition Every day of delay dooms more ALS patients. No words can convey how HORRIFIC the suffering is while we wait on the FDA #NUROWN survival data: UNPRECEDENTED in ALS history & UNAVAILABLE at ADCOMM. Survival data: #GoldStandard for FDA approvals. As @MartyMakary said, 'gold standard science & common sense, guide this FDA Five-Year Survival: 100% in NurOwn EAP vs. 20% in ALS natural history. All EAP participants (n=10) achieved five-year survival without tracheostomies. Extended Tracheostomy-Free Survival (TFS): 7-year median tracheostomy-free survival (range from 5 to 8.5 years), far surpassing the 2.5- to 3-year median in ALS natural history data. Progression-Free Survival (PFS): When patients received NurOwn, they experienced PFS ranging from a few months up to 17 months. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-petit… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy

@MinoShah @DrMakaryFDA @FDACBER @SecKennedy The data is in, the proof is undeniable. We are dying a little more every day we wait. We deserve this chance. It is more than time to do the right thing and give us accelerated approval and a chance at a better and longer life. #nurownworks for some and some is always enough!!!

.@DrMakaryFDA @FDACBER @SecKennedy, It’s been 56 days since we filed our Citizens' Petition For 156 years, ALS has had ZERO survivors. We now have a therapy, NurOwn, with unprecedented, gold-standard survival data Every day of delay dooms more patients. The suffering is immeasurable. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-petit… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @MartyMakary @SenatorWicker @RepRobinKelly @RepBarragan @DrOzCMS @DrOz @RepKimSchrier @RepLoriTrahan @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy

@aVoice4ALS @SenBillCassidy @MartyMakary @rfk Completely agree with @aVoice4ALS - The FDA and FDACBER needs massive change. They are not listening to patients or the experts. Terminal diseases are not getting access to treatments that will help them.

During confirmation hearings for @MartyMakary & @RFK, please discuss how people with TERMINAL rare diseases desperately need DISRUPTIVE policy change at FDA. Some FDA failures include: 1. Disregarding the opinions of world renown rare disease experts/ clinical trial investigators, biomarker experts who testified in Advisory Committee meetings that drugs worked on their dying patients -- especially egregious when the FDA has no similar expertise in the rare disease. This recently happened in #Duchenne, #ALS & #Amyloidosis with experts from Stanford, Mayo, MassGen, Vanderbilt, Boston U, Barrow, Nationwide Childrens & Cleveland Clinic to name just a few. 2. Disregarding the opinions of rare disease treating physicians who opined that a drug helped their patients live and have a better quality of life. One of many examples: regulations.gov/comment/FDA-20…… 3. Ignoring the testimony, videos & medical records from patients in the trials and expanded access who received therapies and experienced how a drug improved how they "felt, functioned & survived." When patients are dying they know when a drug helps them live. Believe them. youtu.be/vrr3uB1TP4o?si…… 4. Opposing a Conditional Approval pathway like Senators' Braun & Gillibrand's Promising Pathway 2.0 bill. Dying Americans are traveling around the world & spending hundreds of thousands dollars, to take unproven & possibly harmful scam therapies rather than getting therapies in the US & collecting that data to further the science. This bill would accomplish that. braun.senate.gov/news/press-rel…… 5. Ignoring the opinions of top statistical experts wth expertise in rare diseases & Bayesian methodology & instead deferring to FDA statisticians with no peer-reviewed publications. Examples: • Ignoring Harvard's Lee Jen-Wei who has 40+ yrs in practice, 300 peer-reviewed studies & is the 2009 Wilks Memorial Award winner for innovating the "totality of the evidence" methodology. • Failing to allow AdComm testimony from Yale/ MD Anderson's Don Berry, who has 50 yrs experience, 400+ peer-reviewed studies & specializes in Bayesian adaptive trial designs, which minimize sample size while increasing the likelihood of detecting therapeutic benefit. 6. Failing to consider how hetereogeneity effects trial endpoints & p-values then denying efficacy when a drug may work on some but not all. When people are dying, #SomeisEnough. 7. Failing to consider post hoc evidence as "supporting evidence" of a drug's efficacy that has valid confirmation in other aspects of the clinical trial -- despite Dr Pazdur's Oncology Center of Excellence considering similar data. In his 32-page Public Comment, Dr. Ardalan Minokadeh MD gave examples of divisions who had properly used Post Hoc evidence. regulations.gov/comment/FDA-20…… 8. Failing to take into account how flawed, subjective "clinical outcome assessments" are impacting trial endpoints & accurate conclusions in assessing efficacy. 9. Failing to follow both the "plain language" & intent of the bipartisan 21st Century Cures Act sponsored by @RepDianaDeGette & passed in 2016. 10. Failing to recognize #RealWorldEvidence and Patient Reported Outcomes that weren't prespecified despite efforts by Rare Disease Caucus chairs like Democrats @DorisMatsui @SenAmyKlobuchar & GOP @SenatorWicker & physician @RepBradWenstrup. The Duchenne community & Rep Matsui introduced the Benefit Act because the FDA is ignoring RWE & RWD. parentprojectmd.org/advocacy/take-…… 11. Failing to give people with terminal rare diseases a statutory right to request an Advisory Committee meeting. prnewswire.com/news-releases/…… 12. Refusing people dying of Rare Diseases their Constitutional Due Process rights to testify in person at AdComms. 13. Depriving patients at Advisory Committee meetings an adequate opportunity to present the first-hand CLINICAL EVIDENCE from taking the therapy, then failing to have any dialogue about that evidence. Some parents with boys dying of Duchenne were denied a chance to speak. Even for those given a chance to share their evidence, three minutes is not ample time to share seven years of evidence in the fight for your life or your child's life. 14. Failing to ensure its "Fast track" progam works when many drugs still aren't approved a decade later despite evidence of efficacy for YEARS. 15. FDA CBER's failure to use RMAT progam to expedite biologic development and approval. 16. Applying different approval standards, AdComm Questions & Instructions among different FDA divisions such as CBER, CDER & Oncology Center of Excellence. 17. Taking years to draft & implement 10 pg Guidance Documents. 18. Failing to follow statutes & Guidance about exercising "Regulatory Flexibility" in terminal diseases with "critical unmet needs." 19. Failing to act with urgency & triage the underfunded terminal rare, diseases that are killing people. 20. Failing to consider the inhumane risk of Type II errors in heterogeneous terminal diseases. This must be factored into the statutory risk-benefit decision. 21. Failing to exercise flexibility to get drugs in bodies of #Veterans who are dying of diseases they get from their service to our country. militarytimes.com/opinion/commen…… The patient community has tried all mechanisms to change this broken regulatory system for the last decade. Patients wrote OpEds. ALS widow @nicolecimbura wrote an OpEd in the @WSJ about how a slow FDA was delaying and denying access to drugs. wsj.com/articles/a-slo…… Physician and pediatric oncology reseacher Bill Woods wrote how the FDA applied different standards in ALS compared to Oncology: "As a pediatric cancer researcher, I admired the FDA. Then I got ALS." statnews.com/2022/08/16/fda…… We've had 1000s of Congressional calls, meetings, and worked with the #RareDisease Caucus. Patients met with the White House, Domestic Policy Counsel, and FDA Commissioners & Directors under Presidents Obama, Trump & Biden. We passed $500M legislation. People wth ALS and leading researchers spoke at multiple Congressional hearings discussing the need for a new approach to terminal rare diseases. youtu.be/mjDwdMpraxY?si…… We have top political allies who advocated for change including Brian Wallach & Sandra Abrevaya who worked for Barack Obama; health care advocate Ady Barkin, and Dan Tate one of the country's top lobbyists. Ady died of ALS. Dan, Brian & 32k others have ALS and are #DyingWaiting for the FDA. Dying patients like Brian Wallach made documentaries such as the Katie Couric produced No Ordinary Campaign in 2023. amazon.com/Love-Life-No-O…… and Angelina Fanous made #DieTrying on HBO Vice in 2017. youtu.be/arDpeOm2s_4?si…… People battling ALS engaged with the media across the country and NBC affiliate WLWT did multiple stories with anchor @ShereeWLWT. wlwt.com/article/cincin…… We participated in protests, Town Halls, AdComms, PFDDs, public meetings, patient advisory boards & industry meetings. We sent private emails to FDA Commissioners & Directors. We spoke at industry conferences on panels with FDA officials. We started non-profits & Facebook groups, paid for billboards and made our own press releases with pleas for our lives. prnewswire.com/news-releases/…… No one has listened. I've cried too many tears about family & friends who died when drugs could have helped them live. I can't speak for any other Democrats but I will work with anyone of any party who will help us in the fight for our loved ones' lives. @SenBillCassidy

The FDA is responsible for ensuring that medical advancements are safe and available to benefit patients, and that our food supply is likewise safe. I’m interested in hearing how Doctor Marty Makary will accomplish this.

Objective confirmation that #NurOwnWorks in #ALS. @BrainstormCell is presenting a poster at the NEALS Conference highlighting EAP data. Poster confirms that people in EAP who received NurOwn mesenchymal #stemcell treatment had a beneficial impact -- just as the people in the P3 trial & EAP have been sharing for YEARS. NfL BIOMARKER DATA IN EAP 1. NfL ≈ stabilized in people who rcvd #NurOwn in P3 & EAP 2. Four of six people on NurOwn in P3 had actual decreases in NfL, possibly indicating the importance of early dosing for optimal impact. 3. Six people on NurOwn in P3 had avg DECREASE of: 4% at end of P3 ... 27% DECREASE at end of EAP1 ... and 36% DECREASE at end of EAP2, possibly demonstratng a dose-dependent response. More NurOwn = More impact. 4. In contrast, people who were on placebo had 37% INCREASE in NfL at end of P3, but only 17% INCREASE at end of EAP1 when they rcvd the first 3 doses of NurOwn and then finally a 5% DECREASE at the end of EAP2 after 6 total doses of NurOwn. 5. 9/10 people in EAP ≈ stabilized when they got NurOwn, regardless if they were in the treatment arm in the trial or didn't receive NurOwn until EAP. 6. Decreases in NfL continued during gap in treatment periods between P3, EAP1 and EAP2, pehaps indicating durability of treatment effect. EAP SURVIVAL DATA 1. Survival in EAP was 5.5 months longer than PRO-ACT control group. 46.6 months in EAP vs 41.1 in PRO-ACT control group (p=0.03) 2. Survival data was not distinguished between those who rcvd NurOwn in P3 & EAP versus those who didn't receive it until EAP. 3. At the last available visit, 9 EAP participants were alive. One had pre-planned euthanasia. Survival was assessed at the date of the last ALSFRS-R score -- not today. 4. What's not shared in the poster: 6/10 people in the EAP are still alive today. None trached. ⬇️You can watch the AdComm testmony of trial PI Tony Windebank and the people in EAP at this link. The objective data shared in the poster confirms what the #RealWorldEvidence that they shared with the FDA at the AdComm in Sept 2023. Since that time 6k people hae #DiedWaiting. youtu.be/vrr3uB1TP4o?si… #ALS #EndALS #MND

Approving #Nurown is not only the right thing to do - it is the moral thing to do #NurownWorks @US_FDA @DrCaliff_FDA @DrBumpusFDA @AmmarAlChalabi @DrOzdinler @aVoice4ALS @iamalsorg @alsnewstoday @alsadvocacy @bsw5020 @heavy_sara @crayne128 @BrielmaierL

@FDACBER Another new day that ALS patients suffer because you won’t listen to the experts, don’t care about real world evidence, don’t support the 21st century cures act, won’t use your power of regulatory flexibility, or accelerated approval to help us. #NurOwnworksforsome #ALSisFATAL

Find out what's new in #biologics today. fda.gov/vaccines-blood…

Agree. The other thing that is unfair is the FDA failed to approve a #stemcell treatment for people with #ALS -- despite having evidence that it slowed progression, improved function & survival for the 1st time in the 160+ yr history of this 100% terminal disease. Imagine a drug helping you live, but your govt is blocking you from getting it. That is not #Freedom. The FDA's actions are contrary to Congressional intent in the 21st Century Cures Act. They are also paternalistic & patently inhumane -- just like the GOP's actions taking health care decisions away from women. @KamalaHarris: your mom Shyamala was a cancer researcher. She knew the importance of helping people with incurable diseases. Now we need you to carry on her legacy. 🔹Help people with terminal #RareDiseases get #DrugsinBodies 🔹Fund late stage grants for innovative treatments for Rare Diseases just like it did Moderna's mRNA vaccine 🔹Establish a Rare Disease Center of Excellence at the FDA just like the Cancer COE transformed by Dr. Pazdur 🔹Support @DorisMatsui's Benefit Act & mandate the FDA consider #RealWorldEvidence like the clinical observations of Mayo's Dr Windebank below 🔹Support @SenGillibrand's & @SenatorBraun's Promising Pathway (#S4426) & @RepDianaDeGette's Cures 2.0 to establish a new regulatory pathways for terminal rare diseases. Obama passed ACA. Bush changed HIV. Biden has the Cancer Moonshot & ARPA-H. Please be the 1st President to to prioritize rare diseases. Please believe dying patients when they tell you that a drug is helping them live. #NurOwn works. Our regulatory law doesn't. We can show you the proof. We need your support. People are #DyingWaiting. #MyBodyMyChoice @amyklobuchar @RepAnnaEshoo @SenMarkKelly @RubenGallego @SenAmyKlobuchar @SenTinaSmith @RepSchakowsky @RepLBR @RepMikeQuigley @ElissaSlotkin @JohnFetterman @TeamPelosi @SenSchumer @RepAngieCraig @hakeemjeffries @MayorGiles @gregstantonaz @DrAmishShah @KamalaHQ @TheDemocrats @harris_wins @KamalaForAZ @KamalaforNC @KamalaforPA @KamalaForMI @KamalaForWI @KamalaforFL @KamalaForNevada @Tim_Walz

@NaderAlAyaseh @DrCaliff_FDA

@BourbonSteep @BrainstormCell Sorry to hear. It’s a shame the treatment is not widely available, it could have easily been if the corrupt @FDACBER @US_FDA showed flexibility it is supposed to under current statutes. #NurOwn @RepLBR @RepDanGoldman @RepMikeQuigley @RepAnnaEshoo @SenatorBraun

If we believe in #MyBodyMyChoice for women, surely we must also believe in humanity for terminally ill patients. We don't need the FDA telling people with 100% terminal diseases like #ALS that it won't approve a #stemcell treatment when they & their world class neurologists testified that it halted their lethal progression & helped some regain lost function -- the 1st time this has happened in the 160 year history of the disease. @Tim_Walz -- MAYO is in your Congressional district! Believe them! #NurOwnWorks In #rarediseases, our regulatory system doesn't. 32,000 People with ALS are #DyingWaiting #Freedom #MindYourOwnDamnBusiness #Kamala2024 #KamalaWalz2024 #EndALS @VP @KamalaHQ @KamalaHarris @POTUS @SecBecerra @neeratanden @amyklobuchar @SenAmyKlobuchar @DorisMatsui @RepDianaDeGette @RepAnnaEshoo @TeamPelosi @TinaSmithMN @RepBarragan @janschakowsky @RepAngieCraig @RepLBR @SenAlexPadilla @CedricRichmond @SenSchumer @SenGillibrand @RepMikeQuigley @maddow @NicolleDWallace @SRuhle @11thHour @morningmika @AliciaOnMSNBC @clairecmc @jonstewart @TheDailyShow @DanaBashCNN @kaitlancollins @GStephanopoulos @ThisWeekABC @SymoneDSanders @MichaelSteele @JoeNBC @WillieGeist @Yamiche

@Cylebo How about a decent sized EAP at each trial site? The community desperately needs this!

$BCLI #NurOwn #ALS Phase 3B Trial preparations are progressing well. We're meticulously laying the groundwork for a successful launch. #BetterTogether Your support fuels our determination to make a difference.

@anneek10s Couldn’t agree with you more. The athletes are amazing and I can barely write at all and can’t use my walker anymore because it’s too much pressure on my shoulder. So now it’s all wheelchair.

All kidding aside I can’t even put into words the depth of despair watching incredible Olympic athletes when I’m struggling to hold my toothbrush. It’s just so sad. #ALSsucks

@anneek10s I’ve heard of people going, but I haven’t heard of any successes yet.

Anyone have experience with the South Korea “Nurown”? Learn More About NurOwn™, A Breakthrough Stem Cell Therapy for Chronic Neurologic Conditions - Springfield Neurology & Functional Medicine Doctor & Treatment: SHN functionalmedicine.doctor/learn-more-abo…

@NaderAlAyaseh @US_FDA #NurOwnWorks. Let pALS have this treatment. It can keep them #StrongEnoughLongEnough

People with #ALS need #acure #Nurown NOW Not 5 years from now. @US_FDA