LogicalValue retweeted
The @FDA has put rare disease patients at the center of its public agenda this week. Senior leadership appeared at National Organization for Rare Disorders summit & yesterday the agency held a major public meeting on rare diseases. I welcome that commitment. With the November 22 decision date for deramiocel approaching, I want to see it reach the families living with Duchenne muscular dystrophy.
I believe FDA should approve deramiocel for an appropriately defined indication focused on preserving upper limb function, with honest labeling, appropriate safety precautions, & continued study. I want to explain why this matters so much to me.
To be transparent, I worked full time at @Capricor Therapeutics $CAPR FOR 2 years (2015-2017 - that's how long this process takes) & helped initiate its muscular dystrophy program. I began cell therapy research as a teenager & have spent decades working on cell biology, regenerative medicine, & therapeutic development for pediatric rare diseases. Over the years I have stayed in touch with patients (some nearly every day), parents, and advocates connected to this program. I have heard their hopes, their frustrations, and their concerns about what another delay could mean. Those conversations stay with you.
For a boy or young man with Duchenne who has already lost the ability to walk, preserving arm and hand function can mean continuing to feed himself, lift a drink, drive a power wheelchair, or bring a hand to his face. These abilities shape how much of his day he can manage independently and how often he must ask someone else for help. A change that looks modest on a clinical scale can matter enormously when it affects something a person does every day. Patients and families have essential expertise in explaining its value. FDA heard those priorities at the advisory committee meeting. Their testimony should help shape how the agency evaluates clinical meaning, acceptable risk, and the consequences of waiting.
There is a scientific basis for taking deramiocel seriously. The program includes two randomized, blinded, placebo-controlled trials. The published HOPE-2 and HOPE-3 studies reported benefits in upper limb function. In the newer extension findings, patients initially assigned to placebo showed slower decline after switching to deramiocel. Noting that the extension was open label and lacked a concurrent placebo group, I nevertheless find the pattern across these studies compelling enough to support a serious case for approval.
FDA’s July review raised some scientific questions, and the additional data and analyses in the amended application should be evaluated directly against those concerns. The amended application now focuses on upper limb function. That claim deserves a careful assessment of the evidence supporting it. Uncertainty about cardiac benefit remains, and the label should accurately reflect what has been established.
This is where senior FDA leadership can make its commitment to rare disease patients consequential. Ensure that the full amended evidence receives a rigorous review informed by Duchenne specialists, treating physicians, and the people living with this disease. Resolve the remaining issues as directly and promptly as possible. Evaluate the treatment against a clearly defined indication and the applicable approval standard.
The agency’s new rare disease initiative recognizes that evidence must be evaluated in the context of the disease, the patient population, and what can realistically be studied. I agree. That principle should inform applications already under review as well as future development programs. Families waiting today should have the benefit of that thinking.
As a physician, I also want the consequences of delay included in the assessment. Another study may answer an important question. It also takes time (many many years) from patients living with progressive disease. A decision to wait carries consequences for the person who needs help eating next year when he could feed himself this year. That possibility deserves serious consideration alongside potential uncertainties of treatment.
Approval would allow eligible patients, their families, and their physicians to discuss whether deramiocel makes sense for them. They could weigh the potential benefit, the infusion burden, the known risks, and whatever uncertainty might remain. An accurate label and a careful medical discussion would help them make that choice. FDA has an essential responsibility to establish that a treatment meets the standard for approval. Patients and physicians then have their own responsibilities in deciding whether to use it. I want a regulatory system that performs its role rigorously and gives those decisions room to happen when the evidence supports them. Putting American patients first requires that entire process to work.
To Acting Commissioner @FDA_KyleD, CBER Director Karim Mikhail, and the deramiocel review team: please use the remaining review period to pursue a scientifically defensible approval for the indication the evidence supports. I believe the published findings and additional follow-up warrant that effort. I believe preserving upper limb function deserves substantial weight. And I believe the families who have participated in these studies and spoken so openly about their lives deserve a decision that fully accounts for what they stand to gain and what continued waiting may cost them.
I want the boys and young men whose families I have heard from over these years to have the opportunity to discuss this treatment with their physicians while they still have function to preserve. FDA’s renewed commitment to rare disease patients gives me hope that this can happen. I am asking its leadership to do everything the science and the law allow to make it possible.
Image: From @CDCgov DMD Resources page
LogicalValue retweeted
❤️ Yesterday, I had the incredible honor of attending and speaking at the @FDA 's Rare Disease Framework Public Meeting in Silver Spring, MD.
There was something truly special about being in that room. You could see and feel the excitement, hope, and passion coming from the rare disease community. But what moved me just as much was witnessing what I believe to be a genuine passion and commitment from the current FDA administration to making this initiative successful.
For those who may not understand why this is so significant, I've spent nearly two decades advocating for the HD community. I've witnessed patients and families pour their hearts into sharing their experiences, only to feel dismissed, disregarded, or unheard.
I've watched previous FDA leadership make public remarks about our community that I found deeply disparaging. I've helped fight to ensure that the voices of tens of thousands of patients and families, represented through two petitions carrying more than 50,000 signatures, would be heard. And I've experienced the heartbreak and frustration of feeling like those voices simply didn't matter.
Experiences like that can make it incredibly difficult to trust the very system you're fighting so hard to work with.
But yesterday felt different. I didn't just hear promises about innovation, flexibility, and putting patients first. I saw people who appeared genuinely invested in making those things a reality. I felt a willingness to listen, to engage, and to recognize that the rare disease community deserves better.
There's someone I especially want to recognize - Amy Rick. ❤️
In 2025, I attended a Critical Path Institute (C-Path) conference where I had the honor of speaking on a panel.
I also remember sitting in the audience listening to Amy Rick speak about the Rare Disease Innovation Hub. Her passion, her vision, and her determination to change how we approach rare disease drug development were unmistakable. I remember thinking, THIS is the kind of person we need fighting for our communities. I became a huge fan of Amy that day.
Amy has been championing progress for the rare disease community for years, navigating challenges and barriers while continuing to push for meaningful change. I didn't have the opportunity to really speak with her yesterday, but she did take a moment in passing to tell me I had done a good job with my presentation. While she probably has no idea how much I admire and respect her, that small interaction meant a great deal to me.
Amy, I hope you know that your passion, persistence, and dedication have not gone unnoticed. THANK YOU.
And to the current FDA leadership, thank you for embracing this initiative and demonstrating what I believe is a genuine commitment to moving it forward. Your enthusiasm, engagement, and willingness to listen have given me renewed hope and a reason to believe that meaningful change really is possible.
#RareDisease #HuntingtonsDisease #FDA #RareDiseaseFramework #TimeMatters @FDA_KyleD @FDADrugs @fdacder
RT @Christina4HD: @laurencurehd, I am incredibly proud of you. It takes extraordinary courage to stand before the FDA and make the most pai…
Cell and gene therapies can change what’s possible for patients living with rare and severe diseases. Cost shouldn’t put that possibility out of reach.
CMS is expanding our Cell and Gene Therapy Access Model to help more Medicaid patients access these life-changing treatments. Learn more about the CGT 2.0 Model: go.cms.gov/CGT
LogicalValue retweeted
❤️ I HAVE SOME EXCITING NEWS TO SHARE!
I am incredibly honored and excited to announce that I've been selected to speak in person at the FDA's Rare Disease Framework Public Meeting on October 28th in Silver Spring, Maryland!
This meeting is especially meaningful because it focuses on the FDA's new initiative and recently released white paper, A New Framework for Rare Disease Review.
For someone who has spent so many years advocating for the Huntington's disease community, this opportunity means more to me than I can adequately put into words.
I'm not just an advocate. I'm gene-positive, someone living with the reality of a progressive, rare brain disease. I've watched this disease take so much from people I love, and I know firsthand what is at stake when we talk about treatment development, regulatory decisions, and the time patients have left.
I've spent nearly two decades fighting for a seat at the table. On October 28th, I'll have the privilege of using that seat to help shape a future where the patient voice is not an afterthought, but a foundation of the process.
And that means everything to me. ❤️
The FDA's new Rare Disease Framework Initiative represents an opportunity to rethink how treatments for rare diseases are developed and reviewed, recognizing that rare diseases present unique challenges that traditional approaches don't always adequately address.
It's an opportunity to discuss what evidence is realistically attainable, how we can improve consistency and predictability in regulatory decisions, and how patients can have a meaningful role throughout the process.
But here's the part I really want everyone to hear:
📣 YOU HAVE A SEAT AT THIS TABLE, TOO!
The FDA is inviting public comments on this initiative, and this is an opportunity for patients, caregivers, families, advocates, researchers, and everyone impacted by rare diseases to provide input.
You don't need a medical degree. You don't need to understand every detail of regulatory science.
💪Your lived experience IS expertise. And your voice deserves to be heard. 💙
📣 HOW TO GET INVOLVED:
📝 Submit a public comment:
regulations.gov/docket/FDA-2…
Docket #: FDA-2026-N-12214
📅 Deadline to submit comments: December 11, 2026
📖 Read the FDA's new white paper and learn about the initiative:
fda.gov/industry/fda-rare-di…
💻 Learn about the October 28th public meeting and how to attend virtually:
fda.gov/news-events/fda-meet…
I'm excited. I'm humbled. And I'm incredibly grateful for this opportunity.
But more than anything, I'm hopeful about what we can accomplish when patients are genuinely included in shaping the systems that determine our futures.
We have an opportunity to make a difference. Let's not waste it.
Because for those of us living with progressive rare diseases, every day matters. Disease progression can cause irreversible damage. And what we stand to lose isn't just time - it's our independence, our abilities, precious moments with our families, and the futures we're fighting so hard to protect.
❤️ Time Matters. And so do our voices.
#RareDisease #HuntingtonsDisease #FDA #PatientAdvocacy #PatientVoice #TimeMatters #RareDiseaseResearch #RareDiseaseFramework
LogicalValue retweeted
***long idea***
Multibagger long idea $BETR.CN
Now before you guys come at me for being bullish a pink sheet name, they literally closed $100M raise led by Deep Track with RA, Perceptive, Marshall Wace, Coastlands, plus Adage, Woodline, Squadron, Stonepine. How often do you see that many big names in a CSE microcap.
Asset is BETR001 = BOL148, a non hallucinogenic, non controlled LSD analog. No trip means no clinic sit, no REMS, and potential at home dosing. That's a different commercial model to the psych names. And if you missed the psych names well just like $ANL after $eras and $rvmd had run here is one with good r/r. BTW it’s lower right now than what all the big names paid $0.25..
Human signal: Karst 2010 in Cephalalgia, refractory cluster headache pts, 3 doses over 10 days. Cycles broken or sharply reduced, remissions lasting mos, no BP or HR changes. Tiny open label set, but it's the exact molecule in humans.
Valuation: I have it at ~726M shs incl PFWs at ~C$0.225, ~$140M FD with warrants and converts, ~$90M net cash. EV ~$50M. At a time where psych names are in the billions.
It will take some time to play out but if you’re patient: IND 1Q27, then Ph1a in healthy volunteers, then Ph1b in cluster and migraine run in parallel, then ph2 in both. Proceeds are earmarked through ph2.
What gets you to 5 to 10x? clean ph1, ph2 cluster data showing cycle breaking, and broader US access over time. Again that is a a 2 to 3 yr data path, not a 12 mo trade. But worth a punt.
And like any investment theres risks with these names Ceruvia (private) is running the same molecule and is ahead. IP sits on a 1950s compound. CSE liquidity is thin. There's overhang from C$0.06 converts and C$0.10 warrants. The team is tiny. So size small and know the risks. Again pink sheet and you HAVE time to get in before any major moves so no fomo pls.
Disclaimer: I am long , not financial advice.
LogicalValue retweeted
Proud to support the #FDA’s #RareDisease Framework Initiative. This game-changing approach recognizes unique challenges faced by rare disease patients & commits to integrating patient voices, supporting innovative science, & modernizing clinical trials. #HD
@mahesh_shenai, Although we have never met, I addressed Dr. Mallika Mundkur at the @FDA's CNPV public meeting, whom I understand is your wife.
After reading nearly 20 of your posts concerning Huntington’s disease (HD) and AMT-130, I have to ask: Are you evaluating the totality of the evidence, or predominantly searching for reasons to discount it?
You repeatedly express hope that you will someday treat Huntington’s disease patients. But how much direct experience have you had with our community? Have you spent meaningful time with symptomatic patients across disease stages, witnessed their progressive neurological decline, or spoken with families about their risk tolerance?
I ask because I have.
HD has devastated three generations of my family. My grandmother died after years of suffering. Her chorea was so severe that she wore padded mittens for nearly four years to prevent her involuntary movements from dislodging her feeding tube.
I was nearly my daughter’s age when my grandmother developed symptoms. Now both my daughter and son are watching their grandfather and aunt experience the same disease. My father is in the middle stages of Huntington’s disease, while my aunt is further along in its devastating progression. As a mother, I live with the painful reality that my children may one day witness this same deterioration in me—or, even more heartbreaking, experience it themselves.
What I witnessed as a little girl, my children are witnessing today. Different generations, the same devastating cycle we are desperately trying to break.
The psychological consequences are equally profound. A fellow Michigander and HD advocate recently lost her father to suicide. Another Michigan chapter board member nearly lost her niece, and several HD friends have lost loved ones to suicide. Suicide risk among people with Huntington’s disease is substantially higher than in the general population.
Why? Imagine caring for someone you love while witnessing years of relentless neurological deterioration, all while knowing you could face the same fate. And even if you escape inheriting the mutation, you may carry the lifelong burden of survivor’s guilt as those you love continue to suffer. This is the profound, multigenerational trauma of Huntington’s disease.
More than 40,000 Americans live with HD, and over 200,000 are at risk. Those figures do not include the countless caregivers and relatives whose lives are also affected.
Your anecdote about a surgeon with another movement disorder reinventing his professional life was particularly revealing. When I read it, I immediately questioned your firsthand understanding of HD.
HD does not afford patients the luxury of simply reinventing their lives. It progressively damages the brain circuits responsible for cognition, judgment, behavior, movement, and independence. Symptoms can worsen over 10–25 years, gradually taking away the ability to work, communicate, maintain relationships, swallow, walk, talk, and care for oneself.
The ability to reinvent one's life requires precisely the neurological capacities HD progressively destroys. HD takes away the ability to meaningfully participate in life altogether.
The 2024 HD Symptoms and Treatment Impact Survey found that nearly three-quarters of respondents would accept treatment risks for five years without progression, while approximately 35% would accept risks for even three years.
Three more years raising children. Five more years working, communicating, remembering, and living independently. More birthdays, graduations, and time with family.
Then consider the economic consequences of inaction. Over 10–25 years, HD families will face neurologists, psychiatrists, hospitalizations, expensive chorea and psychiatric medications, physical and occupational therapy, speech and swallowing therapy, mobility equipment, home modifications, specialized residential care, and eventually around-the-clock assistance.
HD removes two people from the workforce: the patient and the family member who becomes their caregiver. Families lose wages, insurance benefits, retirement savings, and years of financial security. Many conventional care facilities lack the specialized neurological and behavioral resources required for HD, leaving families with limited options.
These costs accumulate across households, employers, Medicare, Medicaid, disability programs, and society.
When evaluating the risks and costs of treatment, why is the cost of inaction so often treated as though it were zero?
If AMT-130 meaningfully preserves independence, the potential economic and societal benefits deserve consideration.
You have described yourself as one of approximately 80 U.S. neurosurgeons with expertise relevant to this procedure. If AMT-130 does not meet your personal threshold for clinical adoption, that is your judgment. But your threshold is not the FDA's regulatory standard, nor should it determine the choices available to every informed patient and qualified physician.
Other neurosurgeons may recognize the broader significance of this investigational gene therapy. If its clinical benefits are confirmed, AMT-130 could help transform how we approach inherited neurodegenerative disorders beyond HD.
Regarding surgical risk, stereotactic neurosurgery has been performed in humans since 1947. Trial experience suggests the procedure has generally been tolerated.
But the question is not whether neurosurgery carries risk. It does. The question is whether those risks are acceptable relative to the potential benefit for patients facing a progressive, fatal disease without an approved disease-modifying treatment.
Your professional risks and our personal risks may look entirely different.
For you, this is a discussion of procedural complications, evidence, and professional responsibility. For us, it is progressive neurological deterioration, dependency, and premature death.
The reported AMT-130 findings, including favorable Total Functional Capacity trajectories compared with external controls, deserve serious consideration. TFC measures employment, financial management, domestic responsibilities, daily living, and care requirements. It measures the independence HD progressively takes away.
Scientific skepticism is essential, but it must be applied symmetrically.
Our community has also been wounded by regulatory rhetoric. @VPrasadMDMPH, your wife’s former boss, recently compared patients pursuing unproven treatments to a drowning person reaching for a blade. Regardless of the intended scientific warning, such language risks reducing informed, deeply personal benefit-risk decisions to acts of desperation, overlooking the devastating consequences patients and families face without treatment.
Our willingness to accept uncertainty is not desperation. It reflects a rational understanding of what HD will otherwise take from us.
Imagine watching someone you love progressively lose the ability to think, communicate, walk, swallow, or even recognize the people they love most. Now imagine living with the knowledge that the same devastating disease could one day affect you, your children, your siblings, or generations of your extended family. This is the reality of Huntington’s disease.
What would a few additional years together mean? What would you do to break the cycle?
I am not asking you to abandon scientific standards. I am asking you to recognize that the consequences of waiting are as real as the risks of treatment.
The absence of treatment is not the absence of risk. It is continued disease progression, escalating dependency, enormous psychological and economic suffering, and irreversible neurological loss.
You may see an investigational neurosurgical procedure that has not met your preferred evidentiary threshold. We see a potential opportunity to preserve the ability to think, communicate, work, love, and remain present in our families' lives.
We are asking for scientific judgment that accounts for the full reality of Huntington’s disease.
P.S. Go Blue! 〽️💙💛
#HuntingtonsDisease
#RareDisease
I have done my absolute best to be honest and transparent with everyone. I genuinely believe in this treatment - a big part of that is because I have seen that it works with my own eyes. Lived experience goes a long way when it comes to being confident something is working, and to personally know some of the participants and know how they are doing now vs before - for me, I have no doubt to the efficacy of the high dose.
LogicalValue retweeted
Congratulations to Karim Mikhail on his appointment as Director of CBER.
I read his post carefully. One line belongs at the front of every review:
“The scientific record tells you what a therapy does. It cannot tell you what waiting for the treatment costs.”
I have sat with families whose disease has no approved treatment. For them a review clock is far more than a metric. It is time they may not have. He is right to put that sentence first. It decides how the rest of the job gets done.
Every regulatory decision carries two risks: the risk of approving a product, and the risk of not approving it. Both are real. Both land on real people. Only one of them makes headlines.
It is easy to say no when you fear tomorrow’s story about a side effect. We will never see the headline about the child who died, the patient who went blind, or the parent who ran out of time because a promising therapy never reached them. For rare and fatal diseases with no other hope, that silence is not neutrality. It is a cost.
This is not an argument for lowering the bar. The FDA must still keep products off the market that carry real risk and offer no meaningful benefit. Inspections matter. Data integrity matters. Informed consent matters. Post-approval commitments and rigorous safety monitoring are how you give desperate patients a chance without abandoning the scientific method.
Rigor and urgency are not opposites. Rigor is what makes speed worth anything. Speed without rigor is recklessness. Rigor that ignores the patient running out of time is a quieter failure — easier to defend in a memo, and just as final for the family.
Where traditional gold-standard trials cannot be done — too few patients, too rapid a decline, a first-in-class mechanism that will never fill a conventional control arm — the honest answer is not “come back in ten years.” Use the best evidence that can actually be generated. Put the uncertainty in plain language. Require the monitoring that protects the next patient.
Then trust American physicians and American patients to decide. The FDA’s job is to make sure the science is sound and the information is complete. Then let patients and doctors do what is best for them.
The FDA’s only client is the American patient. Full stop.
That is why Advisory Committees matter. Open them. Staff them with people who actually treat the disease. Disclose conflicts out loud, under penalty of perjury, before another word is spoken. Give complex science more than a truncated day. Let patients speak. A modest functional gain that looks unimpressive on a slide can be the difference between a life lived and a life lost.
I have enormous respect for the career scientists inside CBER (I have gotten to know many including at other FDA centers) The work on both sides of the desk is harder than the other side believes.
Leadership’s job is to give them a north star that is patient-first, America-first, and innovation-first at the same time — and then to have their backs when they apply it honestly.
“Frontier science only matters when it reaches patients.” Congratulations to Karim. The families who do not have ten years need CBER to get this right.
Weigh both risks. Tell the truth about uncertainty. Protect people from empty products. Do not protect process at the expense of the only person the agency exists to serve.
While risk-benefit analysis is essential, for many serious diseases we also need risk-vs-risk analysis:
The risk of approving a therapy… versus the risk of not approving it.
It’s easy to say “no” when you fear tomorrow’s headlines about a tragic side effect.
We will never see headlines about the patient who died because the drug that could have saved them never reached them.
Let’s keep our focus on patients — not just p-values. ❤️
LogicalValue retweeted
A BLA for a potential first-ever disease-modifying therapy for Huntington’s disease (HD) is moving forward.
More than 200,000 Americans are at risk. HD represents an extraordinary unmet medical need.
A fatal disease. No approved treatment that slows progression. Families who cannot afford more delay.
@FDA, is the CNPV program still moving forward?
This is exactly the kind of urgency the program was created to address.
💙💜
#HuntingtonsDisease
"accelerate access to lifesaving treatments" $QURE
A pantheon of superstar candidates made choosing our next FDA Commissioner one of the toughest decisions of my tenure at HHS. I’m confident President Trump and I chose the best person for the job: Dr. Heidi Overton.
Heidi brings exceptional judgment, professionalism, discipline, and an unwavering commitment to the American people. I have seen her challenge assumptions, demand excellence, and turn bold ideas into meaningful results.
At the @FDA, she will make our food supply safer, modernize drug approvals, unleash American medical innovation, accelerate access to lifesaving treatments, bring greater transparency and accountability to the agency, and restore Gold Standard Science as the foundation of every decision.
Congratulations, Heidi. I look forward to working with you to restore trust in the FDA and Make America Healthy Again. 🇺🇸
LogicalValue retweeted
As I’ve said many times, @FDA @FDAadcomms are critical, especially when it’s a new MOA or there are any questions regarding the efficacy or safety.
However, as we’ve seen, it’s essential to have subject matter experts on the panel, not just in the audience.
For rare diseases, that can be tough because many of the experts have conflicts (have consulted for the sponsor or a competitor, as it’s a small community). In those cases, they should be invited on, albeit with strict ethics disclosures that they personally read aloud when they introduce themselves.
The public would also benefit from having briefing documents made available far longer than 2 days before the AdComm, so that they have time to digest & prepare, and even provide written input ahead of the meeting.
Finally, in cases where there’s simply too much to review and discuss in 8 hours (less including lunch & breaks), we should consider longer AdComms.
I acknowledge they take an enormous amount of preparation, manpower & expense, but it’s an investment worth making.
It was remarkable to watch the experienced melanoma doctors on the panel dismantle the FDA presentation, based on their clinical knowledge and experience. It's really why these adcomms are so valuable. The FDA needs to embrace them, schedule more.
Also, having panel participants who have subject matter expertise makes a huge difference. Melanoma doc debating melanoma data. And no, that's not putting a thumb on the scale bc the melanoma docs on the panel today expressed many of the concerns and agreed with the FDA on the study flaws. One melanoma doc / panelists voted no.
The FDA needs to liberalize its conflict of interest rules to allow smart, experienced clinicians to participate in adcomms.
LogicalValue retweeted
Thank you @FDA_KyleD and @US_FDA for hosting today’s meeting with Huntington’s disease organizations.
I hope this meeting becomes a meaningful step toward advancing promising therapies for Huntington’s disease with the urgency patients and families deserve.
HDSA thanks Acting FDA Commissioner Diamantas and staff for taking the time to meet with the Huntington's disease community.
#HDSA #HuntingtonsDisease #RareDisease #FDA
i think this panel is a great example of what fda panels are supposed to do. the fda presenter was quite young, and as talented and diligent young people do, focused on lots of very detailed shortcomings of replimune's trial, which led to the review team's general feeling they couldn't rely on the data. eg things like if u biopsy a melanoma lesion it could be counted as a response, or a lit review showing a wide range of response to pd1, or the details of how independent radiologists scored lesions. the clinician panelists focused on very different things. things like the number of complete responses, or even if u discount the controversial cases the response rates were still close to 30%, and their real world experience plus familiarity with the literature on how low response rates are in pd1 refractory patients. it is a great example of the value of leadership and experience. karim did a great thing by holding the panel, and hopefully future new fda leadership will similarly be able to guide talented but less experienced fda staff.
