Posted On: 06/20/2014 12:45:04 PM
Post# of 30066
Gerald's tweets just now:
I have received a number of questions related to the size of the market for Wolfram's Syndrome in the United States and worldwide.
Wolfram's is a pediatric condition that causes blindness, deafness, type-1 diabetes and neurodegeneration in the brain.
We previously est. approx. 300 patients worldwide, however in discussions with the Snow Foundation, it is estimated to be btw 10,000-30,000.
It is estimated there are between 500 and 1500 patients in the US, which would qualify Wolfram's as an orphan indication.
Data produced by Dr. Urano and confirmed by the U. of Helsinki indicates MANF protects beta cells in animal models of Type-1 Diabetes
The University of Miami Bascom Eye Research Institute has shown MANF protects retinal cells, including those affects in Wolfram, from death.
The models used by Bascom are models that are characterised by endoplasmic reticulum stress, thereby correlating with Wolfram's biology.
Dr. Urano and the Company believe this new collaboration may effectively treat the key unmet need of blindness in Wolfram's patients.
Given the increase of our understanding of the number of Wolfram's patients (30x - 100x), the revenue potential has increased dramatically.
Needless to say, if the study is effective, we believe the NPV of MANF will increase substantially, while also reducing its risk profile.
We will give more in depth analysis in the near future, as additional data becomes available on MANF systemic delivery for diabetes.
I have received a number of questions related to the size of the market for Wolfram's Syndrome in the United States and worldwide.
Wolfram's is a pediatric condition that causes blindness, deafness, type-1 diabetes and neurodegeneration in the brain.
We previously est. approx. 300 patients worldwide, however in discussions with the Snow Foundation, it is estimated to be btw 10,000-30,000.
It is estimated there are between 500 and 1500 patients in the US, which would qualify Wolfram's as an orphan indication.
Data produced by Dr. Urano and confirmed by the U. of Helsinki indicates MANF protects beta cells in animal models of Type-1 Diabetes
The University of Miami Bascom Eye Research Institute has shown MANF protects retinal cells, including those affects in Wolfram, from death.
The models used by Bascom are models that are characterised by endoplasmic reticulum stress, thereby correlating with Wolfram's biology.
Dr. Urano and the Company believe this new collaboration may effectively treat the key unmet need of blindness in Wolfram's patients.
Given the increase of our understanding of the number of Wolfram's patients (30x - 100x), the revenue potential has increased dramatically.
Needless to say, if the study is effective, we believe the NPV of MANF will increase substantially, while also reducing its risk profile.
We will give more in depth analysis in the near future, as additional data becomes available on MANF systemic delivery for diabetes.
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