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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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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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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
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Mission Therapeutics Appoints Dr Suhail Nurbhai as Chief Medical Officer

The Pharma Data

Dr Nurbhai has more than 25 years of experience and a strong track record in the strategic and operational leadership of all phases of clinical research and development at companies across Europe and the US. The Company is managed by a team with broad international, commercial and clinical-science experience.

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

The Premier Consulting Blog

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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Analysis Life Sciences Thank You What We Expect the FDA to do in February and March 2024 (Updated)

Agency IQ

EPA March 1 FDA Budget FDA’s budget authority will expire as of midnight on March 1 POLITICO March 7 Guidance Expiration FDA’s Guidance on Assessing Covid-19-related symptoms in clinical trials of products intended to treat the disease expires.

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Analysis Life Sciences Thank You What We Expect the FDA to do in March and April 2024

Agency IQ

District Court March 7 Guidance Expiration FDA’s Guidance on Assessing Covid-19-related symptoms in clinical trials of products intended to treat the disease expires.

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