MortenSS

[JOHN BOLARIS @JohnBolaris]

The FDA needs to approve deramiocel .. the clinical studies and the voices of those suffering from DMD makes it overwhelming clear that it works @FDADrugs

[Elijah Stacy @Elijahjstacy]

Grateful to the outstanding team at BioSpace, a truly excellent publication, for the opportunity to share my perspective on the upcoming FDA decision regarding Capricor’s deramiocel! https://t.co/8HD45XnSiz

[Capricor Therapeutics @Capricor]

This weekend at the American Heart Association Scientific Sessions 2026, Dr. Jonathan Soslow of Vanderbilt received the Outstanding Research Award in Pediatric Cardiology for his presentation of the HOPE-3 cardiac results for Deramiocel in Duchenne muscular dystrophy. Congratulations, Dr. Soslow, and thank you to the patients, families and investigators who made this research possible.
Read the press release: https://t.co/ImKWORpWQW
#AHA26 #Duchenne #DMD #ScientificSessions26 #Deramiocel $CAPR

[MortenSS @MSchieller69609]

@adamfeuerstein Adam Baldstein

[MortenSS @MSchieller69609]

@adamfeuerstein @A_May_MD @adamfeuerstein no one cares about your opinion.

[Houman David Hemmati, MD, PhD @houmanhemmati]

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

[NoMonkeyBusiness @NoMonkeyBusine4]

You just approved Anktiva for bladder cancer papillary only which had a PDUFA date of January 7,2027 (three months early), but you can't find it within your hearts to approve Deramiocel, a RARE disease drug with unmet need with beautiful statistics (and which the FDA has mistreated in the past), which has a PDUFA date of November 22?
The DMD boys are getting more and more hopeless every day.
I truly hope the FDA is turning over another leaf, but I'm thinking somebody over there has a bee in his/her bonnet.
@SecKennedy
@FDA_KyleD
@US_FDA
@FDADrugs
@FDACBER
@POTUS
@realdonaldtrump
@elonmusk
@RobertKennedyJr
@SenRonJohnson
@SusieWiles
@SusieWiles47
@LauraLoomer
@DC_Draino
@IvankaTrump
@MelaniaTrump
@ElijahStacy
@Capricor

[Capricor Therapeutics @Capricor]

Capricor today announced new 24-month data from the HOPE-3 open-label extension, presented last week in a late-breaking poster at the 2026 World Muscle Society Congress in Hiroshima, Japan.
Patients who began Deramiocel after a year on placebo experienced a 76% reduction in their rate of upper limb decline compared with their own prior year. At 24 months, both treatment groups had declined less than natural history predicts.
Read the full press release: https://t.co/VcbosxMWgL

#Duchenne #DMD #WMS2026 #Deramiocel $CAPR

[MortenSS @MSchieller69609]

@adamfeuerstein You are talking shit. How much did you get paid? Delayed study, shows people are living longer.

[MortenSS @MSchieller69609]

@adamfeuerstein @Biohazard3737 How much were you paid to write an 'article' about Sellas?

[MortenSS @MSchieller69609]

@adamfeuerstein @Biohazard3737 Adam FUDstein

[NoMonkeyBusiness @NoMonkeyBusine4]

The @FDA obviously does not give one damn about rare diseases, even though THEY published new guidelines in February, 2026 (that's this year) in dealing with them from a regulatory standpoint. Why would you even THINK about staffing an Advisory Committee hearing with professionals who do not treat the subject rare disease on a daily basis? It happened - the one and only hope for Duchenne Muscular Dystrophy, Deramiocel, was railroaded by professionals who know nothing about it. Do heart surgeons operate on the brain? If not, why not? Here's an excellent article explaining the situation:
https://t.co/Aisji0LA28

Our government is morphing from mere negligence to intentional ........ and downright EVIL. The DMD boys deserve better.

@US_FDA
@POTUS
@realdonaldtrump
@elonmusk
@RobertKennedyJr
@SenRonJohnson
@SusieWiles
@SusieWiles47
@LauraLoomer
@DC_Draino
@IvankaTrump
@MelaniaTrump
@ElijahStacy
@Capricor

[MortenSS @MSchieller69609]

@FDA You know you need to approve, which saves muscle and lives.

[The Lancet @TheLancet]

Bloodstream-delivered cell therapy slows muscle decline in young people with Duchenne muscular dystrophy, phase 3 trial finds.

Explore the research 👉 https://t.co/cCXDL550TO

[MortenSS @MSchieller69609]

Let Them live @FDA

[MortenSS @MSchieller69609]

@nyahphengsitthy @skye_witley She can start with helping the DMD community

[MortenSS @MSchieller69609]

@adamfeuerstein Apparently a 'journalist' knows better than The Lancet and Capricor.

Go look at stocks that can regenerate hair instead

[MortenSS @MSchieller69609]

@adamfeuerstein @StocksGoUppp What an idiot.

[Elijah Stacy @Elijahjstacy]

Just got off the phone with Congresswoman Diana Harshbarger’s office. She has been briefed on the most pressing concern facing the DMD community and understands what’s at stake.

Congresswoman Harshbarger is Vice Chair of the Health Subcommittee and recently introduced the Right to Try for Individualized Treatments Act in the House.

KEEP HOPE ALIVE! 🇺🇸

[Elijah Stacy @Elijahjstacy]

“Speak up for those who cannot speak for themselves; ensure justice for those being crushed.” — Proverbs 31:8

[Mary B Schanck @MaryBschanck]

$CAPR Enough with the “more trials” talk. HOPE-3 was a pivotal Phase 3 trial in DMD, with 106 patients, and it met its primary endpoint, showing a 54% slowing of upper-limb functional decline versus placebo. The results were also published in The Lancet following independent peer review.

The real issue now is the regulatory interpretation of the statistical analysis, particularly around the SAP and the cardiac endpoint.

Capricor’s position is that SAP 3.0 was the prespecified analysis plan, and the company continues to believe the existing dataset supports approval.

So from the bull case: stop moving the goalposts and work with the existing HOPE-3 data. The question is whether the FDA can reach an agreement with Capricor on the appropriate statistical framework and move toward approval of Deramiocel.

HOPE-3 produced meaningful clinical results. The Lancet publication adds independent peer-reviewed validation. Now let the FDA finish the job. 🔥🧬

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