@alsctr.bsky.social
Reposted
Reposted
The FDA's case study for the Tofersen approval makes clear NFL is a viable surrogate marker for ALL ALS cases. We can stop saying "ALS has no biomarkers" - not true. Now we need to find drugs that match tofersens dramatic reduction in NFL. t.co/fKqt6P2WRH Thanks @alsctr.bsky.social for the tip!
https://www.fda.gov/media/186135/download
t.co
September 18, 2025 at 4:21 PM
The FDA's case study for the Tofersen approval makes clear NFL is a viable surrogate marker for ALL ALS cases. We can stop saying "ALS has no biomarkers" - not true. Now we need to find drugs that match tofersens dramatic reduction in NFL. t.co/fKqt6P2WRH Thanks @alsctr.bsky.social for the tip!
But it worked for people who wear purple socks on Fridays and have pet cats. Approve it. 🐈
As someone once explained post hoc analyses --
You can't take out the people something didn't work for and then say, "It works."
You can't take out the people something didn't work for and then say, "It works."
September 18, 2025 at 3:33 PM
But it worked for people who wear purple socks on Fridays and have pet cats. Approve it. 🐈
So very sad that he missed the Emmys. My heart is with Eric and his family.
deadline.com/2025/09/emmy...
deadline.com/2025/09/emmy...
Eric Dane Misses Emmys’ Planned ‘Grey’s Anatomy’ Reunion; Fellow Alum Jesse Williams Presents Solo
Eric Dane Misses Emmys' 'Grey's Anatomy' reunion with fellow alum Jesse Williams presenting solo and no tribute to ABC drama.
deadline.com
September 18, 2025 at 3:29 PM
So very sad that he missed the Emmys. My heart is with Eric and his family.
deadline.com/2025/09/emmy...
deadline.com/2025/09/emmy...
Reposted
Something ALS orgs seem never to be able to figure out —how to distinguish your own work without denigrating the work of others.
September 5, 2025 at 7:51 PM
Something ALS orgs seem never to be able to figure out —how to distinguish your own work without denigrating the work of others.
Reposted
RWE from EAPs will be valuable only if a sponsor establishes a protocol for reporting data. This business of writing down a score when you think of it and expecting FDA to be compelled is nuts.
September 6, 2025 at 6:40 PM
RWE from EAPs will be valuable only if a sponsor establishes a protocol for reporting data. This business of writing down a score when you think of it and expecting FDA to be compelled is nuts.
Reposted
Random people representing themselves allow for those hosting meetings to get around accountability and institutional knowledge .
Collaboration without inclusion of diverse voices is an echo chamber of bias.
There is a valuable role for independent voices in patient and disease advocacy. Sometimes in ALS Land we're skipping the difficult conversations in order to get to inter-organizational priorities that everyone with organizational ties is comfy with. Collaboration should not require heads-in-sand.
August 25, 2025 at 3:30 AM
Random people representing themselves allow for those hosting meetings to get around accountability and institutional knowledge .
Collaboration without inclusion of diverse voices is an echo chamber of bias.
There is a valuable role for independent voices in patient and disease advocacy. Sometimes in ALS Land we're skipping the difficult conversations in order to get to inter-organizational priorities that everyone with organizational ties is comfy with. Collaboration should not require heads-in-sand.
August 25, 2025 at 2:36 AM
Collaboration without inclusion of diverse voices is an echo chamber of bias.
Reposted
Shameful. I'm certain they didn't consult the amazing folks at the Clinical Center's Department of Bioethics on this one - not that they should have needed help figuring out how wrong this is.
HHS has now confirmed this
August 8, 2025 at 10:10 PM
Shameful. I'm certain they didn't consult the amazing folks at the Clinical Center's Department of Bioethics on this one - not that they should have needed help figuring out how wrong this is.
The longer I am in ALS the more I cannot unsee.
The disease is a terror. The politics and people only complicate it more.
💔
The disease is a terror. The politics and people only complicate it more.
💔
July 30, 2025 at 2:02 PM
The longer I am in ALS the more I cannot unsee.
The disease is a terror. The politics and people only complicate it more.
💔
The disease is a terror. The politics and people only complicate it more.
💔
Reposted
I had the privilege of participating in my first #allals #preventals visit yesterday @UCSF! I appreciated the professional and cordial work of coordinator Amy and investigator Dr Rosow. My first experience with handheld dynamometry, much quicker than an emg.
July 30, 2025 at 12:45 PM
I had the privilege of participating in my first #allals #preventals visit yesterday @UCSF! I appreciated the professional and cordial work of coordinator Amy and investigator Dr Rosow. My first experience with handheld dynamometry, much quicker than an emg.
Do we know of many cancer drugs approved only on post hoc? I get confused by fuzzy references to how easy it is to approve cancer drugs and no data shared to back that up…
I have seen well thought out critiques of oncology trial designs, but the approvals for those drugs still met some endpoint.
I have seen well thought out critiques of oncology trial designs, but the approvals for those drugs still met some endpoint.
July 15, 2025 at 5:22 PM
Do we know of many cancer drugs approved only on post hoc? I get confused by fuzzy references to how easy it is to approve cancer drugs and no data shared to back that up…
I have seen well thought out critiques of oncology trial designs, but the approvals for those drugs still met some endpoint.
I have seen well thought out critiques of oncology trial designs, but the approvals for those drugs still met some endpoint.
Reposted
What ever happened to #BetterTogether ? Iykyk.
July 11, 2025 at 1:17 PM
What ever happened to #BetterTogether ? Iykyk.
Remembering today that ALS doesn’t stop for holidays. 💔
July 4, 2025 at 1:32 PM
Remembering today that ALS doesn’t stop for holidays. 💔
Reposted
How I wish we had a better grasp of what is and isn;t going n w ALS projects...
Despite resumption of NIH grant reviews, research funding gap grew www.statnews.com/2025/06/27/d... via @statnews.com
Despite resumption of NIH grant reviews, research funding gap grew www.statnews.com/2025/06/27/d... via @statnews.com
Despite resumption of NIH grant reviews, research funding gap grew
NIH has made no progress in narrowing the funding gap created by the freeze on grant approvals despite resuming grant review meetings
www.statnews.com
June 28, 2025 at 10:10 AM
How I wish we had a better grasp of what is and isn;t going n w ALS projects...
Despite resumption of NIH grant reviews, research funding gap grew www.statnews.com/2025/06/27/d... via @statnews.com
Despite resumption of NIH grant reviews, research funding gap grew www.statnews.com/2025/06/27/d... via @statnews.com
AI is becoming inescapable.
It’s great and it isn’t great. Either way it’s very convincing.
It’s great and it isn’t great. Either way it’s very convincing.
June 28, 2025 at 11:30 PM
AI is becoming inescapable.
It’s great and it isn’t great. Either way it’s very convincing.
It’s great and it isn’t great. Either way it’s very convincing.
Reposted
The two worst feelings in the world are not having a diagnosis and having a diagnosis
June 7, 2025 at 8:12 PM
The two worst feelings in the world are not having a diagnosis and having a diagnosis
Reposted
Every Kamala voter in America watching Trump and Musk slapfight because electing the smart Black lady was too much to ask.
June 6, 2025 at 1:44 AM
Every Kamala voter in America watching Trump and Musk slapfight because electing the smart Black lady was too much to ask.
This.
Important Qs for any clinical trial in ALS Land --
1. Do you have the funding to finish the trial?
2. Do you have the funding to allow volunteers to stay on the therapy if they want?
1. Do you have the funding to finish the trial?
2. Do you have the funding to allow volunteers to stay on the therapy if they want?
May 24, 2025 at 4:44 AM
This.
Reposted
For those who can't get into clinical trials, EAP is your route to investigational therapies. If people tell you FDA is the problem, don't believe them.
May 22, 2025 at 10:37 AM
For those who can't get into clinical trials, EAP is your route to investigational therapies. If people tell you FDA is the problem, don't believe them.
Reposted
And are there any people with ALS in this picture?
May 6, 2025 at 11:31 AM
And are there any people with ALS in this picture?
Reposted
Call your senators
Well the NIH has cancelled the Women's Health Initiative, the largest study of women in history
It has been running continuously since 1991 and has provided massive key knowledge about diseases in women
Unreal
www.science.org/content/arti...
It has been running continuously since 1991 and has provided massive key knowledge about diseases in women
Unreal
www.science.org/content/arti...
NIH cancels its first and largest study centered on women
The Women’s Health Initiative has produced numerous influential findings
www.science.org
April 23, 2025 at 1:53 PM
Call your senators
Research on one genetic type of ALS moves us all forward. If the goal is to find subtypes that will respond to drugs designed for them, then we need to be ok with that actually happening.
April 17, 2025 at 4:18 PM
Research on one genetic type of ALS moves us all forward. If the goal is to find subtypes that will respond to drugs designed for them, then we need to be ok with that actually happening.
Well that was concise. For the wordier but no more effective explanation… www.facebook.com/share/p/15yi...
April 14, 2025 at 4:57 PM
Well that was concise. For the wordier but no more effective explanation… www.facebook.com/share/p/15yi...