𝔼𝕕 ℂ𝕠𝕝𝕝𝕚𝕟𝕤

If you are an FDA employee under pressure from the commissioner’s incompetent leadership, I want to hear from you. To submit a disclosure to my office, please email Auchincloss.Oversight@mail.house.gov

We are deeply grateful to everyone helping build momentum for HDSA’s Day of Action. When our community comes together with urgency and purpose, our collective voice becomes impossible to ignore. Visit HDSA.org/HDdayofaction #HDSADayOfAction #OneHDSA #HuntingtonsDisease

Exclusive A Bloomfield Hills "mogul" bought a Rolls-Royce Wraith and a $2.4 million mansion after stealing more than $4 million in COVID-19 relief aid, according to records that portray one of the largest pandemic-era frauds in Metro Detroit history detroitnews.com/story/news/loc…

The way the @Royals support @SarahNauser makes me so happy 😭😭😭💙💙💙 #EndALS

@MarkSharkKing x.com/i/status/20414…

@edcollins23 Didn’t see and couldn’t find that post. Just recall Ian and Doug denying knowledge of 2nd death and then trying to give all the “razzle dazzle.” I have liked them both, easier to do so in the good times. But Ingram has always been hard to trust and Ian is a psych major.

$SRPT So do you think an outside hire as new CEO would lift share price more than simply elevating Ian? Guess it depends on who the outside hire would be, but …Thoughts?

A man who uses a specialized wheelchair says a recent American Airlines trip left his chair damaged. clickondetroit.com/news/local/202…

$qure $clpt Spread the word “Many of us want to grow old with our spouses, watch our children grow up and live happy productive lives without the fear of Huntington’s. We want to stop Huntington’s so that no other husband, wife, mother, father, brother, sister or child of HD has to live with this disease in any way, shape form or fashion. With your help, we hope and pray we can make this a possibility.” @RepAuchincloss @SenRonJohnson @SenRickScott @SenGillibrand @WhiteHouse @FrankLuntz @SusieWiles @BeckyQuick @HDSA @HDBuzzFeed @Help4HDI @adamfeuerstein @MartinShkreli @laurencurehd

Heather Thurgood Wilmoth writes that families affected by Huntington's disease need the public's help in urging federal leaders to speed up drug reviews and expand access to potential treatments for what’s described as a "combination of ALS, Parkinson's, and Alzheimer's." supertalk.fm/column-hunting…

@yachmod Kind of amazing how a media entity that seems to exude knowledge about the muscular dystrophy space doesn't even attend the MDA Conference. Just let that sink in for a moment.

$SRPT Suspect we should be hearing soon of discussion with FDA on the PMOs since STAT is starting to rumble. So predictable.

Satellos is testing what could be a game changing treatment for Duchenne and potentially other muscle wasting diseases - (sports related damage too maybe?). The video interview is well worth your time to watch. Since it seems as if I'm constantly being reminded by the current FDA of how great they are doing, how about proving your greatness to the rare disease community with N>1. SAT-3247 seems like an optimal project to provide a springboard to rather than a roadblock. There are thousands of treatable patients that are watching this very closely not to mention their caregivers and doctors. @FDA @SecKennedy @SenatorSlotkin @SenGaryPeters @RepHaleyStevens @MDAorg

Yesterday I posted my follow up podcast interview with @SatellosBio (Nasdaq: MSLE) (TSX: MSCL) CEO Frank Gleeson on SAT-3247 Interim Trial Updates and Upcomin... youtu.be/CpLi7uAs_uM?si… via @YouTube

This wouldn't be directly beneficial in the short term but in the near term I believe that John F Crowley has given some really strong proposals lately. I've got a link to his testimony to the Senate committee last fall below, (he also gave a presentation at MDA last month outlining this - it is definitely worth the watch youtu.be/v7gaWgI8CO4). The crazy thing is that most of the governmental costs would be in creating a more efficient and helpful FDA. Makary speaks about many of these changes in the works all the time. What really ticks me off is that these changes were taking place, albeit slowly, prior to RFK, MM and VP, and many would have been up and running by now if this regime didn't change. We've lost a good year plus from this. On a good note, I feel the institutions that are heavily invested in clinical trials saw this coming and seem to have devoted themselves to developing treatments while collecting overwhelming data sets that will allow for unprecedented analysis using AI. The science is here or very near. ============================== Testimony of BIO President & CEO John F. Crowley before the Senate Health, Education, Labor, and Pensions (HELP) Committee: “The Future of Biotech: Maintaining U.S. Competitiveness and Delivering Lifesaving Cures to Patients” bio.org/letters-testim…

@edcollins23 @MarkSharkKing @Sarepta Ed, thank you for talking me off ledge again. Insurance mechanism doesn’t work for rare diseases. Not big govt fan but when there are times for public good like rare diseases, ought to be a super fund. It would probably lower insurance costs.They need to think out of box on this!

$SRPT @Sarepta Any talk on street how the “education” and site work is going? Remember before all heck broke loose in Q1 2025 EC the promise was to do “hard work” of building out and supporting non-major dosing sites.

@DrMakaryFDA Show me don't tell me. Rare and ultra rare patients and families know more about their own diseases than the people at the FDA do.

We’re lowering drug prices and getting decisions out efficiently.

There's been a lot of industry, political and regulatory discussions, (not just Sarepta), regarding the complexity of getting smaller health clinics on board with basically what is to them a whole new ball of wax as far as the cost and employees needed to do these one time therapies. Each treatment is pretty much based on a single contact for each patient with insurance reimbursement taking its typically slow time. These smaller clinics probably will never be able to handle the up front costs the way our system is set up. I think what is being seen currently where patients are referred to the primary dosing sites is going to continue until more of these therapies get approved. It will definitely limit the number of patients that can get treated near their home which puts additional logistical and monetary strains on families who are already probably strained enough as it is. Pretty much everyone has to do a go fund me to help cover costs. During a presentation at MDA they gave an estimate of $15K of additional expenses/loss of income. I don't know what the solution would look like in the short term outside of maybe helping cover costs for families. Longer term the first step is going to be the FDA giving some real world clarity that they are supportive of these one time high cost treatments, not just talk.

@yachmod @Sarepta Have heard nothing. Except the echoes of Ingram repeating how “brilliant” his team was and how the “launch was brilliant.” Then we learn they were too cocky to teach up the smaller sites. I like Ian, but he was part of this failure and also the LIE re: 2nd death. Brutal.

During the TD Cowen presentation last month Ian discussed the hiring of inside salesforce and contract sales force - this is copy/paste of partial transcript from Investing.Com -link follows below "Ian Estepan, President and COO, Sarepta: They’re just now kind of hitting the ground running. The first wave. Ritu Baral, Analyst, TD Cowen: They’re trained, they’re hired up. Ian Estepan, President and COO, Sarepta: The first wave has been hired. Ritu Baral, Analyst, TD Cowen: Mm-hmm Ian Estepan, President and COO, Sarepta: ... has been trained, but we’re also doing a contract sales force. Ritu Baral, Analyst, TD Cowen: Oh, okay. Ian Estepan, President and COO, Sarepta: ... that we’re looking to expand to also, so about doubling from the number of reps, that we currently have, and they’ll be more focused on, more of the peripheral sites and finding patients. Ritu Baral, Analyst, TD Cowen: Got it. Okay. The 50% more, or the first wave are Sarepta employees, then they’ve hired a more technical detail. Is that fair to say? Ian Estepan, President and COO, Sarepta: Correct. Yeah, that’s fair. Ritu Baral, Analyst, TD Cowen: The doubling is actually this contract force, and you said their focus is... Ian Estepan, President and COO, Sarepta: Is more on the peripheral sites and patient finding. Ritu Baral, Analyst, TD Cowen: The lower volume sites. What sort of patient finding activity? Ian Estepan, President and COO, Sarepta: It’s just a matter of obviously we’ve seen claims for a lot of patients, but that does not necessarily mean that they’re having the conversations with physicians. Ritu Baral, Analyst, TD Cowen: Mm-hmm Ian Estepan, President and COO, Sarepta: ... around exon skip, around gene therapy or exon skipping. It’s getting those patients in front of the physicians and getting them to a referral site, and making sure they have balanced information." ng.investing.com/news/transcrip…

Before and after video, one year post gene therapy. It's working! The first person was treated with Atamyo's ATA-200 gene therapy for LGMD2C at the University of Florida on April 2,2025. instagram.com/reel/DWp6flkjq… @DionFund @LgmdAwareness @speakfoundation @MDAorg @BeckyQuick

Thank you to everyone at @MLBNetwork for airing this special piece. An extremely special message with words from my heart. 🫶🏼 #ForeverRoyal #FountainsUp #ALS

I’m seriously considering doing a series on #HuntingtonsDisease to show what we mean when we say #ProgressionDoesntPause because too many people still don’t fully understand what that looks like. 🧵 (1)

@JenniferHandt @Elijahjstacy s203.q4cdn.com/439234936/file…

@Elijahjstacy Hey Elijah I sure hope you're right! I have not seen this data, can you point me to the source?

This is very encouraging to me and explains some things imo 1) explains why the data visually looked worse than it actually is because small N makes the entire graph go down with one bad patient result 2) exercise seems to really matter imo because FVC you’re always breathing. I do not know what level of physical activity these patients are doing, but I know they’re constantly breathing, which stimulates the muscle. Dominate hand always appears to improve more