Ann: Neuren and Acadia expand global partnership for trofinetide, page-123

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good1234

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17/07/23

12:06

Post #: 68821611
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Originally posted by royalwulff55: ↑

At risk of getting involved with a poster who is clearly trying to bait or too lazy to do their own research or a mixture thereof I'll reply to your 2 points to save others doing so.

1. NEU and Acadia have both repeatedly used figures of 4500 current patients with Rett Sx in US and repeatedly used figures of up to 9000 potential total patients. So potentially as many undiagnosed patients as those with formal diagnosis. While the blood test is "relatively easy" it has to be ordered by a paediatrician and Rett Sx is one of many neurodevelopmental conditions that have an array of similar symptoms. The patients don't wake up one morning and announce they have Rett Sx. With Daybue approval there will now almost certainly be impetus to seek a formal diagnosis and register number will definitely increase - to what is guesswork but if NEU/Acadia suggest 9000 then that seems reasonable given connections they have with physicians who actually treat the condition.

2. There were many reasons given to explain the relatively high dropout. If you read the actual FDA approval report it is well covered. To list a few: in the trial patients had to commence at the treatment dose rather than titrate, couldn't stop aperients and had to attend multiple appointments often interstate for follow up and blood tests in the middle of a COvId pandemic. Clearly the uptake will be superior now that the drug is available and mostly affordable, can be titrated slowly, aperients can be ceased and there is not the strict conditions of trial follow up. I agree with your point that not all patients will respond to trofinetide the rest of your post is IMO garbage.

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I was going to add similar (not bothered with as much detail as you have provided)

Anyway, another point I wanted to add, in addition to the total numbers of Retts patients, was that the thought in general(that I’d heard anecdotally) was that many Retts patients and families suffer in silence because no treatment options were available.

With the positive results from this first available drug to help was that many additional patients would / could present because help was now available.

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Ann: Neuren and Acadia expand global partnership for trofinetide, page-123

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Ann: Neuren and Acadia expand global partnership for trofinetide, page-123

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