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Bridging Gaps in Rare Disease Clinical Trials: Insights from Collaborative Success in Pompe Disease Research

XTalks

Orphan drug development poses many challenges that are unique to rare diseases, including recruitment in small patient populations, variable progression with heterogenous clinical manifestations and limited understanding of disease progression and natural history. Read on to learn key lessons in rare disease drug development.

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Solving the Global Clinical Trial Gap: The Case for Partnering with Multisite Lab Services

XTalks

As the research and medical community learns more about population variances in disease outcomes, whether as a result of regional genetic profiles, socioeconomic factors, or environmental inputs, it’s become necessary to ensure that clinical research trials include broad swaths of global participants. billion in 2019.

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What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers

FDA Law Blog

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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International Women’s Day 2024: A Spotlight on 5 Women’s Health Webinars with Xtalks

XTalks

Experts discuss the therapeutic landscape, treatment gaps, regulatory considerations and clinical trial strategies. Breast Cancer Multiomics: Unified Insights in Tumor Heterogeneity This on-demand webinar introduces a cutting-edge single-cell multiomics method designed for garnering detailed genetic insights into breast cancer.

Genetics 115
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Hope in sight with Apellis’ pegcetacoplan OAKS/DERBY results for GA

pharmaphorum

Asked to elucidate the specifics of what the study had found, Dr Wykoff’s positivity over the “large, global clinical trial” was palpable; certainly, he is deeply passionate about his specialist area. “To To dive into specifics, […] OAKS and DERBY were identical trials. The specifics of an exciting prospect. Dr Charles C.

Genetics 105
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Why Do Black Patients Fare Worse With Blood Cancer Than Whites?

The Pharma Data

And even when Black people with AML have the same access to treatment as white patients, their survival is shorter — something genetic differences might explain. But even when patients of both races received the same treatments, Black patients’ outcomes were worse, clinical trial data showed.

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Immunotherapies could target aggressive pancreatic cancer, research indicates

The Pharma Data

Our findings could help to pick out those patients most likely to benefit from immunotherapy – and we’re keen to translate our work into clinical trials to test the benefit of different immunotherapeutic strategies to tackle this hard-to-treat form of pancreatic cancer.”. Source link.