TEARS of HOPE

U.S. FDA made history by using #biomarkers & #AcceleratedApproval to change future of #SOD1 #ALS just as it has #cancer .

Thx @Ionis & @Biogen for your #ASO technology & Tim Miller of Washington U. Med for your leadership

#Qalsody means Cassandra can be here for her kids!

Earlier this year, we partnered with I Am Biotech, The Rare Disease Company Coalition, and a handful of companies to commission research from VitalTransformation to document how proposed changes in #AcceleratedApproval would impact rare disease. View the findings: bit.ly/3b2bi58

Why Joe Biden Nominee Dr. Robert Califf Would Be A Pro-Patient FDA Head ibtimes.com/why-joe-biden-…

A combination of CSF #Biomarkers demonstrate high accuracy in predicting clinical outcomes.  Can you say #AcceleratedApproval ?

New research from VitalTransformation finds up to 66% of accelerated approval treatments could be at risk of not being developed if the pathway was not available, resulting in up to 3.6mil people losing access to treatments. Learn more: buff.ly/3aSa38r

#OneRareVoice

Biogen announced cut to programs from #ALS & #ALZ pipelines bc “lower quality of success”

Drugs that work on SOME in vastly heterogeneous diseases like ALS must be approved via #AcceleratedApproval to advance science & spur investment

fiercebiotech.com/biotech/biogen…

FierceBiotech

#AAADV19 is starting with @AjeFashoyin talking about U.S. FDA approval processes. Here’s a primer on the #acceleratedapproval pathway

'Although it is important that some types of #research are incentivised by #AcceleratedApproval mechanisms, this does not mean the threshold for safe and reliable evidence should be any lower'. #cancer #medicines
The risks of hype for hope.. The Lancet Oncology

Important webinar about #biomarkers

Biomarkers can be used as 'surrogate endpoints' for sponsors seeking #AcceleratedApproval of their #ALS therapies

WHEN: Thursday at 5 pm ET

⬇️Register:
partners.zoom.us/webinar/regist…

axeALSFoundation No More Excuses ALS Watchdogs! I AM ALS Brian Wallach
Lisa's Legacy for ALS

People living with ALS are dying waiting for U.S. FDA to listen to our voices @peterpitts @califf001 #ALS took my running legs but #NurOwn gave me time with family and hope. Cheering from the sidelines is better than the alternative. #LivingProof #FileTheBLA #AcceleratedApproval

I have the right to live! Don't let me die without trying NUROWN.WE DON'T HAVE TIME.NUROWNNOW
#AcceleratedApproval
#NurownWorld
Mary Kay Turner
C Lebovits
David setboun
Ralph Kern
EU Medicines Agency
U.S. FDA
Dr. Stephen M. Hahn
President Biden
European Commission
Matthias Groote
EU Health - #HealthUnion
Stella Kyriakides
Ursula von der Leyen

Kylie died at 27.
Need @NINDS to fund research studying OMICS of many young people who get 'sporadic' #ALS .

Walter J. Koroshetz please make this part of the Strategic Plan. Dr. Robert M. Califf please give them a chance by using #AcceleratedApproval so they dont #DieWaiting

Her ALS Story

30 doses would make me grow old with my pals.
#NurOwn for ALL!
FDA Biologics
#AcceleratedApproval

Simple, fast, and reliable fundraising!

Independence for an #ALS patient
Moving hand for 1st time in 18 mos after 3 doses of #NurOwn in #ExpandedAccess at Mayo Clinic.

Q: What would 30 doses do?
A: Let's find out w #AcceleratedApproval & P4 Post Marketing study

#LivingProof #NurOwn Works #PatientReportedOutcomes

Please join us on 9/1 at 5:00 pm EST for a webinar about biomarkers and the HEALEY ALS Platform Trial. This week's webinar will be hosted by James Berry, MD, MPH. Les Turner ALS Foundation ALS ONE EverythingALS NEALS Consortium Muscular Dystrophy Association

Don't fall for it, Idaho. Ranked Choice Voting is a trojan horse to destroy Idaho. 65% Republican Idaho will turn blue faster than you can count to potato.

withdrawconsent.org/the-gift-of-od…

If FDA Commissioner removes #AcceleratedApproval for #Makena after this week’s hearing - weeks after granting #AcceleratedApproval to Amylyx’s #Relyvrio - the U.S. FDA signals a willingness to lower the entry bar for unproven products in favor of withdrawing them later.

ICYMI, Reshma Ramachandran and Holly Fernandez Lynch wrote in MedPage Today about why U.S. FDA should hold off on #acceleratedapproval and wait for confirmatory trial evidence for Sarepta’s DMD Gene Therapy medpagetoday.com/opinion/second…

I have the right to live! Don't let me die without trying NUROWN.WE DON'T HAVE TIME.NUROWNNOW
#AcceleratedApproval
#NurownWorld
Mary Kay Turner
C Lebovits
David setboun
Ralph Kern
EU Medicines Agency
U.S. FDA
Dr. Stephen M. Hahn
President Biden
European Commission
Matthias Groote
EU Health - #HealthUnion
Stella Kyriakides
Ursula von der Leyen

Dear U.S. FDA, Dr. Patrizia Cavazzoni & Dr. Robert M. Califf this legacy is NOT one you want to pass down. You have the power to change the course of history by #EndTheLegacyApproveTofersen #AcceleratedApproval

🌬Admire the rims that flash at top speed and fabulous driving maneuvers as the fast 1960s wind turbine car hits the wind in your backyard!
Get it👉🏻gardengeer.co/E7822

Dear U.S. FDA, Dr. Patrizia Cavazzoni & Dr. Robert M. Califf, give this precious baseball player more time with their dad. You have the opportunity to prolong the life of those living with SOD1 ALS like Chris Snow by #EndTheLegacyApproveTofersen #AcceleratedApproval #patientreportedoutcomes

CRRIT's Reshma Ramachandran in AP News on the recent scrutiny of the U.S. FDA #acceleratedapproval process 'definitely led to them pausing and taking a different path' but that it may be too early to tell if FDA’s recent actions reflect a longer-term policy shift. apnews.com/article/health…

Sharing my vulnerability- I broke down in tears. Thank God it was in the lunchroom. Thank you to our amazing community, Biogen and U.S. FDA. Today, I have hope #SOD1 #ALS #tofersen

My favorite baseball player got new cleats today and promptly personalized them. ❤️⚾️
#4ALS #LouGehrigDay #SnowyStrong #EndALS

48 years today John Percey the 1st past away from Sod1 ALS 45 and 46 years later his grandsons John Percey 2 nd & Ethan passed away same ALS gene mutation leaving behind two more young families without dads. Approve Toferson on 4/25 Dr. Robert M. Califf please don’t let me lose anymore

#AcceleratedApproval was developed by U.S. FDA in 1992 in response to HIV/AIDS. Cancer patients benefited from 167 accelerated approvals by FDA Oncology with only 17 withdrawals. Great #AACR22 session by Gautam Mehta, MD Vivek Subbiah, MD Diana Bradford Sara Whitlock Dr. Wirth

New reforms to FDA Oncology #AcceleratedApproval offer both opportunities and challenges for oncology drug approval. New commentary with David J. Benjamin, MD
thelancet.com/journals/lanon…

Happy to be selected as #EditorsPick in The Lancet Oncology

Surgery and Cancer Medscape Oncology pharmalot

FDA delays Sarepta's #Duchenne gene therapy decision by a month - More like this pharm2market.app | Sarepta Therapeutics U.S. FDA #Research #GeneTherapy #Celltherapy #AcceleratedApproval #Delay #DrugDiscovery #Biotech endpts.com/fda-delays-sar…

27 year old Kylie died in 2022 of #ALS due to FDA Biologics regulatory RIGIDITY denying safe/effective #Nurown .

Over 160 years that #ALS is 100% fatal & still NO URGENCY @ the U.S. FDA.

Dr. Robert M. Califf Rep. Anna G. Eshoo Senator Mike Braun Jan Schakowsky Rosa DeLauro Brett Guthrie Ken Calvert