Biomarker For Acute Aortic Syndrome May be Especially Important To Evaluate in Connective Tissue Disorders
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Biomarker For Acute Aortic Syndrome May be Especially Important To Evaluate in Connective Tissue Disorders

A new study has demonstrated that plasma desmosine could be an acute aortic syndrome biomarker. This also means it could be a potential diagnostic tool. The findings from this study…

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Study of the Week: Whole Plant Cannabis Succeeds in Rare Childhood Epilepsies Where CBD and Others Fail
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Study of the Week: Whole Plant Cannabis Succeeds in Rare Childhood Epilepsies Where CBD and Others Fail

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

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Xywav for Idiopathic Hypersomnia Earns Orphan Drug Exclusivity
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Xywav for Idiopathic Hypersomnia Earns Orphan Drug Exclusivity

Orphan Drug designation is typically granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases; in the United States, these are conditions affecting under 200,000 Americans. Drug…

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Phase 1 Trial for Primary Biliary Cholangitis Therapy Produces Promising Results
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Phase 1 Trial for Primary Biliary Cholangitis Therapy Produces Promising Results

Albireo Pharma Inc. has just announced the results from their Phase 1 clinical trial for A3907, an ASBT inhibitor. The study produced positive results, achieving its primary and secondary endpoints. Researchers…

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Compassion Corner: Compassionate Use, Expanded Access Part One: The Terminology Is Different But the Unmet Need Is the Same
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Compassion Corner: Compassionate Use, Expanded Access Part One: The Terminology Is Different But the Unmet Need Is the Same

Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…

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“With Mais and Mal:” Meet Two Sisters Changing the Conversation around MID & Life with a Rare Disease (Pt. 2)
Mallory and Maisy with their family. Photo courtesy of Mallory Cyr

“With Mais and Mal:” Meet Two Sisters Changing the Conversation around MID & Life with a Rare Disease (Pt. 2)

In Part 1 of "With Mais and Mal," Patient Worthy spoke with Mallory Cyr about microvillus inclusion disease (MID), Mallory's story, and the process of becoming an advocate. Today, we…

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Whole-Plant Medicinal Cannabis Found to be Superior to CBD as Treatment for Childhood-Onset Epilepsy

A study conducted by Rayyan Zafar and his colleagues from the Imperial College London was recently published in BMJ Paediatrics Open. This investigation evaluated the efficacy of whole-plant cannabis as a treatment for…

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Study Shows New Gene Therapy Technique Successful in Reversing Severe Hearing Loss in Mice
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Study Shows New Gene Therapy Technique Successful in Reversing Severe Hearing Loss in Mice

According to a recent article, a new study from Boston Children’s Hospital shows that a first-of-its-kind gene therapy technique has succeeded in reversing severe hearing loss from a mutation to…

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Acute Hepatic Porphyria Treatment Now Has NICE Approval and Will Soon be Available Through The NHS
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Acute Hepatic Porphyria Treatment Now Has NICE Approval and Will Soon be Available Through The NHS

Givlaari, a treatment for acute hepatic porphyria (AHP), was first approved in Europe back in March of 2020. However, approval doesn't mean immediate access unfortunately. It's been nearly two years…

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