Amyotrophic Lateral Sclerosis Drug Developers Set Their Sights on a New Treatment Target
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Amyotrophic Lateral Sclerosis Drug Developers Set Their Sights on a New Treatment Target

On March 22, 2023, via PRNewswire, the FDA announced that its CNS Drugs Advisory Committee voted unanimously in favor of a potential accelerated approval of tofersen to treat people with…

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Draft Guidance from the FDA Suggests Important Changes to Drug Development
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Draft Guidance from the FDA Suggests Important Changes to Drug Development

On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983).  The Draft Guidance considers…

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For Patients Under Consideration for CAR-T Therapy The Cancer Support Community/Gilda’s Club Has A Free Program

No One Should Face Cancer Alone. That is the mission of the non-profit Cancer Support Community (CSC). It offers emotional support and hope via programs designed for cancer patients, cancer…

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Research!America Discussion with Dr. Mary Pittman: Better Data for More Equitable Research
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Research!America Discussion with Dr. Mary Pittman: Better Data for More Equitable Research

On April 12, 2023, Research!America, a Patient Worthy partner organization, hosted a webinar program titled Better Data for More Equitable Public Health Research. This talk featured Dr. Mary Pittman, President…

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Rare Community Profiles: How Stephanie’s Desmoid Tumor Journey Inspired Her Fight Against Medical Gaslighting
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Rare Community Profiles: How Stephanie’s Desmoid Tumor Journey Inspired Her Fight Against Medical Gaslighting

Rare Community Profiles     Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

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