Thanks for the explanation Endless. Considering you have been on HC since I first joined in 2009, I imagine you have seen your fair share announcements. I assume you are also a successful trader so I was interested to hear your POV.
Agree about the speculation over $$$ going on for years but I don't think that makes NEU any different to other tech companies developing a new product.
I think the announcement was great news and the results are indeed encouraging to sufferers of Rett Syndrome and their families. However my experience of working in the field of neurological disorders and subsequently having a child with one may bias my opinion, hence my interest in hearing your views.
Btw in the world of medical research 'encouraging' is the word given to a positive outcome. The language of scientific research is always measured and conservative, quite the opposite to that used by traders otherwise the announcement would have been titled "Boom".
Where I differ:
- study size reflects the fact that it is a rare disease and therefore much smaller pool to draw from who must also meet strict trial criteria
- in addition to meeting primary and secondary endpoints, there was clinically significant improvement in many other areas which is why "More than 95% of participants who completed the Lavender study elected to roll-over to the Lilac open-label extension study." (the post on NEU titled "Thank you" will give you a carer's insight into the drugs efficacy (those living with the sufferers are best placed to assess the impact of treatment)
- side effects are a feature of many drugs, esp. those used to treat neurological disorders and are often mitigated with other drugs (common practice) and these side effects are easily managed and not detrimental to health. Furthermore adjustments to dosage etc can be made outside a trial setting and thereby reduce the incidence of side effects
hence I believe Trofinetide will very likely get FDA approval.
Also worth noting Rett Syndrome is a disorder and not a disease. At present we don't have any drugs that 'cure' syndromes, only drugs that treat their symptoms which is what Trofinetide does for the sufferers of Rett Syndrome.
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Thanks for the explanation Endless. Considering you have been on...
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