Ken M
3K posts
@kjmark
Chiefs, Yankees and golf fan. Hoping ALS patients get access to needed treatments.
We’re lowering drug prices and getting decisions out efficiently.
George Brett joined #MLBCentral to speak on his bond with Sarah Nauser and her fight against ALS. Learn more here: iamals.org/stories/sarah-…
FDA Commissioner Dr. Marty Makary revealed a major shift at the agency that’s still making waves: “I personally meet one-on-one with reviewers and ask: ‘Are you seeing anything that gives you chills up your spine because it could be such a game-changer?’” He described the FDA’s new proactive approach: - No more passive waiting for applications. - When early data looks “too good to be true,” they call companies directly: “Forget the paperwork — send more data now.” - Fast-track vouchers for decisions in weeks instead of years. - Recent example: gene therapy restored normal hearing in 3 of 12 deaf children (improved in 7) with perfect safety — FDA contacted the company days after NEJM publication. Makary emphasized the FDA is aggressively tracking promising treatments for blindness, deafness, pediatric/rare diseases, cancers — and a “potentially promising” therapy for type 1 diabetes that has reviewers excited. This is the FDA acting like it’s on the front lines of curing disease, not just regulating it. Months later — are you hopeful this new mindset finally delivers real cures, or waiting to see hard results?
How long must hopeless patients wait for a chance at life? It's been 8 months since we filed the #NurOwn Citizens Petition—well past the FDA's 6-month deadline @FDACBER & @VPrasadMDMPH: We have the evidence for accelerated approval for a 100% fatal disease. Stop the silence. 🛑
FDA should move with urgency to address rare diseases.
The FDA has a moral duty to move quickly to get meaningful treatments to patients.
@Coinvo Here's whistleblower Peter Rost (former @pfizer executive) keeping it 💯:
MASSIVE: 🇯🇵 Japanese scientists have used regenerative medicine to restore motor function in paralyzed patients!
I wanted to share that my dear friend Leanne passed away peacefully last week. Her community here meant so much to her, especially her fellow PALS. Thank you for the friendship, kindness & support you gave her, it meant the world to her. ❤️ dignitymemorial.com/en-ca/obituari…
.@DrMakaryFDA @FDACBER @SecKennedy, we MUST remember those who have died/are dying/will die from HORRIFIC 100% FATAL MILITARY SERVICE-RELATED #ALS Navy vet Matt Bellina, ALSFRS-R 21, wheelchair-bound, choked eating, needed BiPAP. After 7 NurOwn doses via Right to Try: +6 pts to 27, stood unassisted (arms-free after 2 yrs), no choking, breathed independently, speech/swallow improved, lung capacity +23%, core/arm strength up! Despite these UNPRECEDENTED benefits, @BellinaMatthew & ALL with ALS have been left to die. @BrainstormCell @Cylebo MUST meet @MartyMakary @VPrasadMDMPH about Accelerated Approval of SAFE & EFFECTIVE #NurOwn We MUST STOP CONDEMNING these heroes to die WITHOUT HOPE. While we delay, veterans are dying. 1 in 6 ALS deaths are veterans – this alone deserves URGENT ACTION from Congress We cannot stand idly while those who served our country are abandoned by the very system meant to protect them. I urge the new @US_FDA leadership to grant #Nurown the Commissioner's National Priority Voucher Program #CNPV Honor our veterans' sacrifices by fighting for their right to life. Veteran organizations & currently healthy veterans MUST join the fight to END MILITARY SERVICE RELATED #ALS. Veterans have a 2X to 10X ⬆️ ALS risk depending on military occupation For veterans with HORRIFIC #ALS today & the MANY who will be diagnosed, we MUST END the CRUEL @FDACBER regulatory RIGIDITY for 100% FATAL #ALS Veterans' voices are among the most powerful in this fight. We MUST END 156 years of hopelessness that disproportionately affects military veterans. HOW MANY MORE HAVE TO BURY LOVED ONES before 100% FATAL ALS (since 1st identified in 1869) is treated with the same URGENCY & regulatory flexibility as cancer treatments? @realDonaldTrump @POTUS @RobertKennedyJr @HASCRepublicans @HASCDemocrats @SASCGOP @SASCDems @HouseVetAffairs @VetAffairsDemson @SVACGOP @SVACDems @BipartisanVets @RepAndyHarrisMD @RepDonBacon @SenatorWicker @DrNealDunnFL2 @RepDanCrenshaw @RepMMM @RepJames @RepGuthrie @RepJackBergman @RepVanOrden @RepKiggans @RepAbeHamadeh @VernBuchanan @SenJimBanks @RogerMarshallMD @sentuberville
America’s veterans safeguard our nation’s birthright of freedom. Because of their strength and courage, America stands safe, strong, and free — forever the bravest and mightiest nation on Earth. Happy Veterans Day to all who served. 🇺🇸
.@DrMakaryFDA @FDACBER @SecKennedy, we MUST remember those who have died/are dying/will die from HORRIFIC 100% FATAL MILITARY SERVICE-RELATED #ALS Navy vet Matt Bellina, ALSFRS-R 21, wheelchair-bound, choked eating, needed BiPAP. After 7 NurOwn doses via Right to Try: +6 pts to 27, stood unassisted (arms-free after 2 yrs), no choking, breathed independently, speech/swallow improved, lung capacity +23%, core/arm strength up! Despite these UNPRECEDENTED benefits, @BellinaMatthew & ALL with ALS have been left to die. @BrainstormCell @Cylebo MUST meet @MartyMakary @VPrasadMDMPH about Accelerated Approval of SAFE & EFFECTIVE #NurOwn We MUST STOP CONDEMNING these heroes to die WITHOUT HOPE. While we delay, veterans are dying. 1 in 6 ALS deaths are veterans – this alone deserves URGENT ACTION from Congress We cannot stand idly while those who served our country are abandoned by the very system meant to protect them. I urge the new @US_FDA leadership to grant #Nurown the Commissioner's National Priority Voucher Program #CNPV Honor our veterans' sacrifices by fighting for their right to life. Veteran organizations & currently healthy veterans MUST join the fight to END MILITARY SERVICE RELATED #ALS. Veterans have a 2X to 10X ⬆️ ALS risk depending on military occupation For veterans with HORRIFIC #ALS today & the MANY who will be diagnosed, we MUST END the CRUEL @FDACBER regulatory RIGIDITY for 100% FATAL #ALS Veterans' voices are among the most powerful in this fight. We MUST END 156 years of hopelessness that disproportionately affects military veterans. HOW MANY MORE HAVE TO BURY LOVED ONES before 100% FATAL ALS (since 1st identified in 1869) is treated with the same URGENCY & regulatory flexibility as cancer treatments? @realDonaldTrump @POTUS @RobertKennedyJr @HASCRepublicans @HASCDemocrats @SASCGOP @SASCDems @HouseVetAffairs @VetAffairsDemson @SVACGOP @SVACDems @BipartisanVets @RepAndyHarrisMD @RepDonBacon @SenatorWicker @DrNealDunnFL2 @RepDanCrenshaw @RepMMM @RepJames @RepGuthrie @RepJackBergman @RepVanOrden @RepKiggans @RepAbeHamadeh @VernBuchanan @SenJimBanks @RogerMarshallMD @sentuberville
