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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.

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

XTalks

With this has come an increase in the size of the global clinical trials market, with an estimated worth of around $46.8 The lab has processed over 25,000 samples, participated in almost 60 clinical trials, and has partnered with over 25 different pharmaceutical companies. trillion, with oncologics accounting for $99.5

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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. 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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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.

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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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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