Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

    Highlights from RAREfest: Cambridge’s Rare Disease Festival

    This weekend Patient Worthy attended RAREfest, an event being held in the UK by the Cambridge Rare Disease Network to raise awareness and promote discussion around rare disease issues. The…

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    Her Rare Condition Revealed his Rare Ability: Amy and Sam’s Service Dog Story
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    Her Rare Condition Revealed his Rare Ability: Amy and Sam’s Service Dog Story

    Amy Dahm, who currently lives in Washington, DC, was working as a U.S. diplomat when she was diagnosed with Cushing’s syndrome. After receiving life-saving treatment from the National Institutes of…

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    $1.4 Million Donated to Help Bring a Gene Therapy For Tay-Sachs Disease to Clinical Trial
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    $1.4 Million Donated to Help Bring a Gene Therapy For Tay-Sachs Disease to Clinical Trial

    A $1.4 million dollar donation to the University of Massachusetts Medical School will help to fund research into a gene therapy for Tay-Sachs disease as it transitions from pre-clinical to…

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    A Device May Reduce the Uncertainty Around Shunt Failure For Patients With Hydrocephalus
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    A Device May Reduce the Uncertainty Around Shunt Failure For Patients With Hydrocephalus

    In a recent news article, the Northwestern Medicine Feinberg School of Medicine has shared details about an experimental device that may have the potential to dramatically improve the treatment of…

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    A Potential Drug For Bronchopulmonary Dysplasia is Ready For Human Trials, Developers Say
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    A Potential Drug For Bronchopulmonary Dysplasia is Ready For Human Trials, Developers Say

    An investigational new drug for bronchopulmonary dysplasia, called CF-MEV-132, has completed pre-clinical in-vivo testing. According to The Cell Factory, who are developing the drug, the potential treatment is now ready…

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    A Recently Published Report Looks into Neuromuscular Disease Care
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    A Recently Published Report Looks into Neuromuscular Disease Care

    A recently published report on neuromuscular disease care outlines the current challenges and future opportunities in diagnosis and treatment. The report, titled ‘Understanding Neuromuscular Disease Care’, was funded by the…

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    The First Patient Has Been Dosed in a Study of a Drug For Idiopathic Pulmonary Fibrosis
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    The First Patient Has Been Dosed in a Study of a Drug For Idiopathic Pulmonary Fibrosis

    The first patient has been dosed in a new clinical trial of an investigational drug for idiopathic pulmonary fibrosis. The drug, called GLPG1205, is being developed by Galapagos, and you…

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    Research Suggests There May be a Link Between Daytime Sleepiness and Attention
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    Research Suggests There May be a Link Between Daytime Sleepiness and Attention

    A study has found that daytime sleepiness and attention may be correlated in people with narcolepsy and hypersomnia, with patients who self-report feeling more sleepy also being more likely to…

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    New Research Suggests Many People Aren’t Aware They Carry BRCA Mutations
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    New Research Suggests Many People Aren’t Aware They Carry BRCA Mutations

    A study has found that more than 80% of participants carrying genes linked to breast cancer didn’t know they had them. The research paper, called ‘Exome Sequencing-Based Screening for BRCA1/2…

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    A New Drug Application Has Been Submitted For a Potential Vascular Ehlers-Danlos Syndrome Treatment
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    A New Drug Application Has Been Submitted For a Potential Vascular Ehlers-Danlos Syndrome Treatment

    Acer Therapeutics has submitted a New Drug Application to the United States Food and Drug Administration on behalf of Edsivo™, their experimental treatment for vascular Ehlers-Danlos syndrome. To find out…

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    A Class of Drugs Called MDM2 Inhibitors May Have the Potential to Benefit Patients With Uveitis
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    A Class of Drugs Called MDM2 Inhibitors May Have the Potential to Benefit Patients With Uveitis

    A study has found that a class of drugs known as MDM2 inhibitors may have the potential to help patients with ocular inflammation uveitis, a serious condition that affects the…

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    New Updates on Studies of Lumasiran in Patients With Primary Hyperoxaluria Type 1
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    New Updates on Studies of Lumasiran in Patients With Primary Hyperoxaluria Type 1

    Alnylam has provided updates about their programs for patients with primary hyperoxaluria type 1 (PH1), a rare condition that affects the kidneys and bladder. As part of their Alnylam Act® program,…

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    A Study Investigated the Effects of Late-Onset Pompe Disease on Patients’ Voices

    Late-onset Pompe disease is associated with many features, one of which is a change to the voice, which may be noticeable in speech and voice quality. To investigate how this…

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    The 2018 Nobel Prize For Medicine Was Awarded to Two Researchers Working on Checkpoint Inhibitors in Cancers
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    The 2018 Nobel Prize For Medicine Was Awarded to Two Researchers Working on Checkpoint Inhibitors in Cancers

    The 2018 Nobel Prize for Medicine was awarded to two researchers working on checkpoint inhibitor therapies for cancer. A source article by AFP, shared by medical express, outlines how checkpoint…

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