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

XTalks

Participant recruitment is challenging, especially in those indications in which there are competing clinical trials necessitating a comprehensive understanding of the disease research ‘landscape’ and targeted outreach and stakeholder collaboration. Sharing patient experiences is also important for gathering real-world insights.

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

pharmaphorum

Having presented the data from the 24-month phase 3 trial at the American Academy of Ophthalmology (AAO) annual meeting, Dr Wykoff set aside some time to go into further detail with pharmaphorum about how the results were received, and what glimmer of hope this might hold for patients with GA.

Genetics 105
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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. The webinar provides insights into a novel method that facilitates detailed genetic analysis of breast cancer, marrying the depth of whole-genome studies with the precision of targeted analysis at the single-cell level.

Genetics 115
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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.

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

The Pharma Data

Bhavana Bhatnagar, an Ohio State University (OSU) cancer specialist and lead author of the second study, “Access can potentially be an issue, in terms of being able to get to tertiary care centers that have clinical trial availability and physicians who have disease-specific expertise in treating some of these rarer cancers.