Draft Guidance from the FDA Suggests Important Changes to Drug Development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Draft Guidance from the FDA Suggests Important Changes to Drug Development

On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983).  The Draft Guidance considers…

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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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The Chan Zuckerberg Initiative is Providing Financial Support for Patient-Centered Rare Disease Research
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The Chan Zuckerberg Initiative is Providing Financial Support for Patient-Centered Rare Disease Research

 In 2003 Ricky Safer was diagnosed with primary sclerosing cholangitis (PSC). Her doctors were not able to tell her much about the origin of her disease. Nor were they able…

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Students Cheer on Peer with Chromosome 2q24 Microdeletion Syndrome as He Heads to a Special Olympics Competition
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Students Cheer on Peer with Chromosome 2q24 Microdeletion Syndrome as He Heads to a Special Olympics Competition

When Alexander Barron (9) was first born, doctors told his parents that he would likely never walk. In fact, said doctors, Alexander would probably not be very independent at all…

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Rare Community Profiles: How Dixie Commits to Supporting her Son and the MSMDS Community
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Rare Community Profiles: How Dixie Commits to Supporting her Son and the MSMDS Community

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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New Guidance from the FDA Intends to Improve Cancer Clinical Trials for Accelerated Approval
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New Guidance from the FDA Intends to Improve Cancer Clinical Trials for Accelerated Approval

According to a press release from the US Food and Drug Administration (FDA), on March 24, 2023, the agency issued a new draft guidance titled Clinical Trial Considerations to Support Accelerated…

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Rare Community Profiles: St. Jude Children’s Research Hospital Opens 45,000 Square Foot “Family Commons” to Support and Offer Comfort to Families
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Rare Community Profiles: St. Jude Children’s Research Hospital Opens 45,000 Square Foot “Family Commons” to Support and Offer Comfort to Families

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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Mutual Trust Between Doctors and Parents of Children with Rare Diseases Means Optimum Care for the Child

The complexity of rare diseases makes communication between adults involved with the care of these children a vital tool. Communication and mutual trust are of the utmost importance. About the…

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Eyedrops Recalled After P. Aeruginosa Infections Killed 3, Injured 65
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Eyedrops Recalled After P. Aeruginosa Infections Killed 3, Injured 65

  Ezri Care Artificial Tears, as well as nine other ophthalmologic brands that create artificial tears or eyedrops, have been linked to dangerous Pseudomonas aeruginosa infections in people across sixteen different…

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Rare Community Profiles: How the #RAREis Global Advocate Grant Supported the E. WE Foundation: A Discussion with Sarita Edwards
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Rare Community Profiles: How the #RAREis Global Advocate Grant Supported the E. WE Foundation: A Discussion with Sarita Edwards

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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New Rare Disease Network Launches in Ireland
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New Rare Disease Network Launches in Ireland

According to a story from sciencex.com, Queen's University Belfast, University College Dublin, and a team of 33 other partners have come together to start the All-Ireland Rare Disease Interdisciplinary Research…

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