Carole Ho
South San Francisco, California, United States
1K followers
500+ connections
Activity
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The use of biomarkers to develop treatments for rare genetic diseases is an essential topic that warrants serious consideration. Learn more on page 1…
The use of biomarkers to develop treatments for rare genetic diseases is an essential topic that warrants serious consideration. Learn more on page 1…
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Read pages 2 and 3 of our “Qualifying Biomarkers to Support Rare Disease Regulatory Pathways” workshop summary to gain a better understanding of…
Read pages 2 and 3 of our “Qualifying Biomarkers to Support Rare Disease Regulatory Pathways” workshop summary to gain a better understanding of…
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And following on quickly after the peer-reviewed article that presenters at this workshop recently published (see my post from earlier today), the…
And following on quickly after the peer-reviewed article that presenters at this workshop recently published (see my post from earlier today), the…
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Publications
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Parallel discovery of Alzheimer's therapeutics.
Science translational medicine
As the prevalence of Alzheimer's disease (AD) grows, so do the costs it imposes on society. Scientific, clinical, and financial interests have focused current drug discovery efforts largely on the single biological pathway that leads to amyloid deposition. This effort has resulted in slow progress and disappointing outcomes. Here, we describe a "portfolio approach" in which multiple distinct drug development projects are undertaken simultaneously. Although a greater upfront investment is…
As the prevalence of Alzheimer's disease (AD) grows, so do the costs it imposes on society. Scientific, clinical, and financial interests have focused current drug discovery efforts largely on the single biological pathway that leads to amyloid deposition. This effort has resulted in slow progress and disappointing outcomes. Here, we describe a "portfolio approach" in which multiple distinct drug development projects are undertaken simultaneously. Although a greater upfront investment is required, the probability of at least one success should be higher with "multiple shots on goal," increasing the efficiency of this undertaking. However, our portfolio simulations show that the risk-adjusted return on investment of parallel discovery is insufficient to attract private-sector funding. Nevertheless, the future cost savings of an effective AD therapy to Medicare and Medicaid far exceed this investment, suggesting that government funding is both essential and financially beneficial.
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Allied forces always make faster progress! This last February, after over a year of planning and engagement, the National MPS Society, in…
Allied forces always make faster progress! This last February, after over a year of planning and engagement, the National MPS Society, in…
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I am so happy to hear this news and I am proud of all the hard work the entire Beam team put in to make this major milestone happen. It's an…
I am so happy to hear this news and I am proud of all the hard work the entire Beam team put in to make this major milestone happen. It's an…
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Don't miss this timely article from BioCentury Inc. featuring insights from our CEO, Emil Kakkis, urgently calling for a new approval pathway for…
Don't miss this timely article from BioCentury Inc. featuring insights from our CEO, Emil Kakkis, urgently calling for a new approval pathway for…
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International Sanfilippo Syndrome Alliance (ISSA) welcomes the news that Ultragenyx has been given the green light to make a Biologics Licence…
International Sanfilippo Syndrome Alliance (ISSA) welcomes the news that Ultragenyx has been given the green light to make a Biologics Licence…
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We are pleased to share the FDA has selected DNL126 (ETV:SGSH), an investigational treatment for MPS IIIA (Sanfilippo syndrome Type A), for…
We are pleased to share the FDA has selected DNL126 (ETV:SGSH), an investigational treatment for MPS IIIA (Sanfilippo syndrome Type A), for…
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May 15 is International MPS Awareness Day and the global MPS community is declaring "It's About Time" for increased awareness, accelerated research…
May 15 is International MPS Awareness Day and the global MPS community is declaring "It's About Time" for increased awareness, accelerated research…
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It was great moderating our important discussion today on the progress and ongoing challenges in rare disease drug development. Thanks to CDER's…
It was great moderating our important discussion today on the progress and ongoing challenges in rare disease drug development. Thanks to CDER's…
Liked by Carole Ho
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Advocacy leaders Matthew Ellinwood, Chief Scientific Officer at the National MPS Society, and Mark Dant, Executive Director of The Ryan Foundation…
Advocacy leaders Matthew Ellinwood, Chief Scientific Officer at the National MPS Society, and Mark Dant, Executive Director of The Ryan Foundation…
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We are thrilled to announce the completion of enrollment for Regimen G of the HEALEY ALS Platform Trial, evaluating DNL343 from Denali Therapeutics.…
We are thrilled to announce the completion of enrollment for Regimen G of the HEALEY ALS Platform Trial, evaluating DNL343 from Denali Therapeutics.…
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I am honored and humbled to be recognized as one of this year's Top 10 HR Professionals by OnCon Icon Awards. I am deeply grateful to my colleagues…
I am honored and humbled to be recognized as one of this year's Top 10 HR Professionals by OnCon Icon Awards. I am deeply grateful to my colleagues…
Liked by Carole Ho
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The widespread adoption of precision medicine is critical to improving outcomes for oncology patients. Through our recently announced…
The widespread adoption of precision medicine is critical to improving outcomes for oncology patients. Through our recently announced…
Liked by Carole Ho
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#ICYMI: All "Qualifying Biomarkers to Support Rare Disease Regulatory Pathways" workshop materials are available on our website. Review the slides…
#ICYMI: All "Qualifying Biomarkers to Support Rare Disease Regulatory Pathways" workshop materials are available on our website. Review the slides…
Liked by Carole Ho
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It was a privilege to participate in the recent Qualifying Biomarkers to Support Rare Disease Regulatory Pathways workshop alongside the National MPS…
It was a privilege to participate in the recent Qualifying Biomarkers to Support Rare Disease Regulatory Pathways workshop alongside the National MPS…
Liked by Carole Ho
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Press Release: Advocacy Groups Join Reagan-Udall Foundation and FDA for Workshop on Developing Biomarkers to Support Accelerated Approval in Rare…
Press Release: Advocacy Groups Join Reagan-Udall Foundation and FDA for Workshop on Developing Biomarkers to Support Accelerated Approval in Rare…
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