Thanks for posting & yes good read. I watched the FDA’s 2024 rare disease presentation on utube. I’d like to add patient voice is also very important. Patients tend to a little more direct & less fluff. Must be due to the death sentence many are facing.
If only our young sr-aGVHD patients could talk & tell of their excruciating, painful, & fearful fight for life. Those parents who spoke at our 2020 ODAC meeting told of the horrible effects of this terrible disease & the affects on their children’s bodies, along with the lifelong affects of having Ruxolitnib which had failed
Mesoblast has been listening all along.
MSB SHs listened to that meeting & the voices of parents & patients will never be forgotten, regardless of any SP.
FDA… I hope they are listening because the data supports this treatment being made available to those who have to fight to live, only to find themselves in a losing battle.
Full credit to Peter Marks for catching up & pulling others along to the reality of modern & innovative medical treatments!
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