Fact Check: Brineura no longer slows CLN2 progression after significant decline in functions.

Fact Check: Brineura no longer slows CLN2 progression after significant decline in functions.

Published June 20, 2025
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VERDICT
Partially True

# Fact Check: "Brineura no longer slows CLN2 progression after significant decline in functions." ## What We Know Cerliponase alfa, marketed as Brine...

Fact Check: "Brineura no longer slows CLN2 progression after significant decline in functions."

What We Know

Cerliponase alfa, marketed as Brineura, is an enzyme replacement therapy approved for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease. Studies have shown that Brineura can significantly slow the decline in motor and language functions in children with CLN2 disease. For instance, a recent analysis of real-world data indicated that ERT-treated patients had a mean rate of motor-language score decline of 0.46 points per 48 weeks compared to 1.88 points for untreated natural history controls, demonstrating a significant difference (p = 0.0003) (source-1).

However, the claim that Brineura "no longer slows CLN2 progression after significant decline in functions" suggests that there is a threshold of decline beyond which the treatment's efficacy diminishes. While some reports indicate that certain patients experience a decline in function despite treatment, the overall evidence suggests that Brineura continues to provide benefits in slowing progression for many patients, even after some functional decline (source-4).

Analysis

The claim oversimplifies the complexities of treatment outcomes for CLN2 disease. The evidence indicates that while some patients may experience a decline in motor functions, Brineura has been shown to slow disease progression effectively in a broader population. The study referenced in the claim shows that ERT-treated patients had a significantly reduced likelihood of experiencing an unreversed decline in motor-language scores compared to untreated controls (source-1).

Moreover, the assertion that Brineura is ineffective after a certain point of functional decline lacks comprehensive support from the available data. The analysis of real-world outcomes suggests that while individual cases may vary, the treatment continues to demonstrate efficacy in slowing progression across a range of functional statuses (source-4).

The reliability of the sources used in this analysis is generally high, as they include peer-reviewed clinical studies and data from reputable clinical registries. However, it is essential to consider that individual patient experiences can differ significantly, and anecdotal reports of treatment failure may not reflect the overall efficacy of the therapy.

Conclusion

The claim that "Brineura no longer slows CLN2 progression after significant decline in functions" is Partially True. While there are instances where patients may not respond as expected after a decline in function, the broader evidence indicates that Brineura continues to slow disease progression for many patients, even after some functional decline. The complexity of treatment outcomes in CLN2 disease necessitates a nuanced understanding of individual patient responses and the overall efficacy of the therapy.

Sources

  1. Real-world clinical outcomes of patients with CLN2 disease treated with ... Link
  2. Brineura® (Cerliponase Alfa) (for Louisiana Only) Link
  3. Fact Check: Brineura no longer effective after certain decline in motor ... Link

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Fact Check: Autistic Non-Verbal Episodes in Marriage: Why Words Vanish Sometimes and What to Do About It Neurodiverse Couples Tuesday, august 12, 2025. Here’s the scene: You’re in the middle of a conversation with your spouse. Maybe the topic is small (“Did you pay the water bill?”) or monumental (“Are we happy?”). And then—without warning—your autistic partner’s voice disappears. No yelling, no slammed doors. Just… gone. You’re left holding the conversational steering wheel while they’ve quietly climbed into the trunk. If you’ve never lived with high-functioning autism, this can be tragically misconstrued as stonewalling or contempt. It isn’t. It’s just neurology pulling the emergency brake. Why This Happens: The Science Without the Lab Coat Smell For autistic adults, losing speech under stress is often a shutdown—a form of nervous system overload that knocks language production offline. Think of it like your phone freezing: all the apps are still there, but none of them open when you tap. Research calls this autistic burnout when it happens in a longer, chronic cycle—linked to masking (Hull et al., 2017; Raymaker et al., 2020). Masking is the art of “performing normal” so well that non-autistic people think you’re fine. The issue is that it eats through your energy reserves like a car idling in traffic with the A/C on full blast (Mantzalas et al., 2022). Eventually, one hard conversation can tip you from functional to frozen. And here’s where couples therapy meets neuroscience: physiological flooding—the body’s fight/flight/freeze switch—is a known relationship killer (Malik et al., 2019; Gottman Institute, 2024). In other words, for some autistic partners, flooding may tend to show up sooner, last longer, and is more likely to pull the plug on speech entirely. 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F., et al. (2023). The lived experience of meltdowns for autistic adults. Autism, 27(7), 1787–1799. https://doi.org/10.1177/13623613221145783 Malik, J., et al. (2019). Emotional flooding in response to negative affect in romantic relationships. Journal of Couple & Relationship Therapy, 18(4), 327–349. https://doi.org/10.1080/15332691.2019.1641188 Gottman Institute. (2024, March 4). Making sure emotional flooding doesn’t capsize your relationship. Retrieved from https://www.gottman.com/blog/making-sure-emotional-flooding-doesnt-capsize-your-relationship/

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Think of it like your phone freezing: all the apps are still there, but none of them open when you tap. Research calls this autistic burnout when it happens in a longer, chronic cycle—linked to masking (Hull et al., 2017; Raymaker et al., 2020). Masking is the art of “performing normal” so well that non-autistic people think you’re fine. The issue is that it eats through your energy reserves like a car idling in traffic with the A/C on full blast (Mantzalas et al., 2022). Eventually, one hard conversation can tip you from functional to frozen. And here’s where couples therapy meets neuroscience: physiological flooding—the body’s fight/flight/freeze switch—is a known relationship killer (Malik et al., 2019; Gottman Institute, 2024). In other words, for some autistic partners, flooding may tend to show up sooner, last longer, and is more likely to pull the plug on speech entirely. The Danger Loop in Marriage Autistic partner goes non-verbal — brain says “nope.” Non-autistic partner reads it as avoidance — brain says “attack.” Pressure increases — “Just say something.” Shutdown deepens — and now you’ve both lost. Do that a few hundred times and you’ll start conflating a physiological response into a moral failing. That’s the real marriage-killer. The Protocol: Three Phases, Zero Guesswork This is where we get practical. You can’t “love away” a temporary shutdown, but you can stop it from turning into World War III. Before: Build the Net Name the state. Agree on a phrase or signal ( I call this a couple code)—such as “words offline,” “shutdown,” a hand over the heart. The point is to make the invisible visible. The Shutdown Card. A literal card that says: I can’t speak right now. Please lower lights, reduce sound, give me X minutes. I promise I will circle back. The Pause Rule. Require a minimum of 20 minutes before resuming any tough talk. Autistic partner may need 90+. Agree ahead of time. Downgrade Kit. the usual gear; earplugs, soft light, weighted blanket, fidget, a quiet room. You know, human decency in object form. Reduce Daily Load. Avoid heavy talks right after work or big social events. Chronic overload makes a nervous shutdown more probable. During: Do Less, Better Autistic Partner: Give the signal. Exit stimulation. Switch channels if possible (text, notes app, yes/no cards). Send a short pre-written message: “Safe, can’t talk, back at 8:15.” Non-Autistic Partner: Acknowledge once—“Got it, I’m with you.” Hold the pause boundary. Lower stimuli. Go regulate your own nervous system—walk, journal, pet the dog. Don’t rehearse comebacks. Both: Avoid sarcasm, interrogation, ultimatums. Nothing lengthens a shutdown like moral outrage. After: Close the Loop Check in: “Are you ready to talk, or should we start in text?” Debrief: Identify triggers and what helped. Solve the actual problem. No conflict gets left to rot in the corner. Spot burnout early. If shutdowns start clustering, it’s time to reduce demands, not double them. How This Isn’t Stonewalling Stonewalling is a choice. Shutdown is a lockout. Stonewalling says, “I won’t talk to you.” Shutdown says, “I can’t talk to you yet, but I will.” The key difference? Repair intention. A shutdown protocol builds that right into the process. The Ten-Minute At-Home Drill Co-create your signal and card. Agree on a pause window. Pack the downgrade kit. Rehearse the exchange (“Got it, I’m with you.”). Check in weekly to tweak the system. Remember, you’re not aiming for zero shutdowns. You’re aiming for shorter, kinder, safer ones. Why This Works Because it matches lived autistic experience (Raymaker et al., 2020; Lewis et al., 2023). Because it honors nervous system limits instead of punishing them (Malik et al., 2019). Because it lets both partners keep their dignity and still solve the problem. 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