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notahackeratllon FDA approves first gene therapy for treatment of genetic hearing loss

This is not linked to MFN pricing, which only impact Praulant…(PCSK9 for hypercholesterolemia)

notahackeratllon FDA approves first gene therapy for treatment of genetic hearing loss

This was not a political decision. These kind of accelerated paths have been available for years for a wide variety of therapies.

notahackeratllon FDA approves first gene therapy for treatment of genetic hearing loss

I would think the time frame is not nearly as long as that… hopefully

notahackeratllon FDA approves first gene therapy for treatment of genetic hearing loss

Consider this from the perspective of a deaf person. While it seems silly from the perspective of a hearing person, a lot of people in the deaf community are concerned that they are viewed as having a problem needing to be fixed, rather than competent, highly functioning people.

notahackeratllon FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss

Yes, some of the vector was likely cleared from the AH and exposed the contra lateral eye

notahackeratllon FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss

Patients with fever induced hearing loss regardless of OTOF mutations were specifically excluded (see inclusion criteria on clinical trials.gov for NCT05788536).

With regard to your point about shedding or systemic exposure. If non functional copies of OTOF in the other tissues expressing the gene were to be replaced by functional copies, what is the concern? How would that negatively impact patients? Doesn’t seem like this would/should be a concern.

Also, mutations don’t have to be teleologically beneficial to occur and persist. They can persist because they are not fatal nor do they impair reproductive competence.