Study of the Week: Scientists Discover a Potential Therapy for an Aggressive Form of Mitochondrial Disease
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Study of the Week: Scientists Discover a Potential Therapy for an Aggressive Form of Mitochondrial Disease

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…

Continue Reading Study of the Week: Scientists Discover a Potential Therapy for an Aggressive Form of Mitochondrial Disease

Editor’s Choice: A New Dystonia Center of Excellence, A Call for Sickle Cell Blood Donors, and a New Treatment Target for Glioblastoma

Happy Friday! A tropical storm system will be traveling up through the mid-Atlantic this weekend, bringing wind and rain. Hunker down! This week, we have stories on Duke University being…

Continue Reading Editor’s Choice: A New Dystonia Center of Excellence, A Call for Sickle Cell Blood Donors, and a New Treatment Target for Glioblastoma
Gene Therapy for Cerebral Adrenoleukodystrophy Earns FDA Accelerated Approval
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Gene Therapy for Cerebral Adrenoleukodystrophy Earns FDA Accelerated Approval

According to a press release, the gene therapy company bluebird bio, inc. has recently announced that its gene therapy elivaldogene autotemcel (marketed as SKYSONA) has received Accelerated Approval status from…

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Editor’s Choice: Charcot-Marie-Tooth Disease Awareness, a Partner’s Accomplishment, and an African Meningitis Vaccine Campaign

Happy Friday! The days are getting shorter and the first cooling winds of autumn have started to blow. This week, we have stories on Charcot-Marie-Tooth Disease Awareness Month, a special…

Continue Reading Editor’s Choice: Charcot-Marie-Tooth Disease Awareness, a Partner’s Accomplishment, and an African Meningitis Vaccine Campaign
Webinar: Improving Classical Homocystinuria Outcomes Through Newborn Screening
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Webinar: Improving Classical Homocystinuria Outcomes Through Newborn Screening

On September 8, 2022, HCU Network America, a Patient Worthy partner organization, hosted a webinar program titled "Classical Homocystinuria: A Journey to Improve Outcomes Through Newborn Screening Methodology." The webinar…

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September 24 is Familial Hypercholesterolemia Awareness Day
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September 24 is Familial Hypercholesterolemia Awareness Day

September 24, 2022 will be recognized as Familial Hypercholesterolemia (FH) Awareness Day, a time to spread awareness about this under-recognized condition among the general public and in the medical field.…

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Lupus Nephritis: Belimumab Alongside Standard Treatment Improves Outcomes in East Asians
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Lupus Nephritis: Belimumab Alongside Standard Treatment Improves Outcomes in East Asians

According to a story published in Healio, a recent study has concluded that a combination of standard treatment and belimumab produces better outcomes for patients of East Asian descent living with…

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Study of the Week: A Device That Can Detect Alzheimer’s 17 Years Before Symptoms Start
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Study of the Week: A Device That Can Detect Alzheimer’s 17 Years Before Symptoms Start

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…

Continue Reading Study of the Week: A Device That Can Detect Alzheimer’s 17 Years Before Symptoms Start

Editor’s Choice: A Biomarker for Early Alzheimer’s Detection, A Teen Living with Usher Syndrome, and A Possible Chagas Disease Treatment

Happy Monday! This week, we have details on a method for detecting Alzheimer's 17 years in advance, the story of a teen living with Usher syndrome, and a possible breakthrough…

Continue Reading Editor’s Choice: A Biomarker for Early Alzheimer’s Detection, A Teen Living with Usher Syndrome, and A Possible Chagas Disease Treatment