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At Long Last, FDA Unveils Plan for Rare Disease Innovation Hub

FDA Law Blog: Drug Discovery

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. By Sarah Wicks & James E.

FDA 105
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Biomarker identification in the realm of rare diseases

Drug Target Review

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

Disease 59
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Antibody Drug Conjugates: windows of opportunity

Drug Target Review

In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. In the ADC trial, the objective response rate (ORR) – defined as the percentage of patients whose disease decreased or disappears – was 42 percent whilst the ORR in the small molecule trial was 12.5

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Women in STEM with Kristina Torfgard

Drug Target Review

I am a pharmacist by training and continued with a PhD in Clinical Pharmacology. However, my goal was all the time to work with drug development in the pharma industry, so I moved on and started that journey in 1992 when I took on a role as Clinical Research Manager at AstraHässle, a mid-size Swedish pharma company.

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In Silico Modeling Unveils a New Era in Rare Disease Drug Development

The Premier Consulting Blog

This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases.

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New strategy for COVID-19 prophylaxis?

The Pharma Data

Gunther Hartmann from the Institute of Clinical Chemistry and Clinical Pharmacology at the University Hospital Bonn, in cooperation with other members of the cluster of excellence ImmunoSensation2 at the University of Bonn, have shown this in mice. SARS-CoV-2 belongs to the genus Betacoronavirus.

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Bristol Myers Squibb Data at the EADV 30th Anniversary Congress Highlight the Growing Body of Evidence on Deucravacitinib and Scientific Advancements for Patients with Serious Dermatologic Diseases

The Pharma Data

Deucravacitinib demonstrated efficacy no matter baseline characteristics, including weight , disease severity and former treatment with biologic or non-biologic therapies. Deucravacitinib is being studied in multiple immune-mediated diseases, including psoriasis, rheumatoid arthritis , lupus and inflammatory bowel disease.

Disease 52