Why Dr. Fiorentino Believes in a Promising Treatment Future for Dermatomyositis
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Why Dr. Fiorentino Believes in a Promising Treatment Future for Dermatomyositis

In some cases, dermatomyositis can be difficult to treat. A variety of treatment options are available, from corticosteroids and immunosuppressants to antimalarials, colchicine, alendronate, and warfarin. But this rare condition…

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New Podcast Episode: The Power of Resiliency, feat. Multiple Myeloma Patient Advocate Keisha Hickson
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New Podcast Episode: The Power of Resiliency, feat. Multiple Myeloma Patient Advocate Keisha Hickson

Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with professional speaker and…

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Positive Results from the Phase 3 Trial of ANX005 in Guillain-Barré Syndrome
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Positive Results from the Phase 3 Trial of ANX005 in Guillain-Barré Syndrome

The long-awaited positive results of Annexon Inc.’s Phase 3 trial investigating ANX005 for Guillain-Barré syndrome (GBS) was presented at the PNS 2024 Annual Meeting. Several leading global GBS experts spoke…

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RNU4-2 Variants Cause Up to 0.4% of Neurodevelopmental Disorders
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RNU4-2 Variants Cause Up to 0.4% of Neurodevelopmental Disorders

In unedited, early-access research published in Nature, a team of researchers explain that an estimated 60% of people with neurodevelopmental disorders remain undiagnosed, despite comprehensive genetic testing options. In many cases, this is…

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Enrollment Complete for Phase 3 Study Evaluating aPAP Treatment Molgramostim
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Enrollment Complete for Phase 3 Study Evaluating aPAP Treatment Molgramostim

Clinical-stage biopharmaceutical company Savara Inc. was founded on a desire to develop innovative therapies for people living with rare respiratory diseases. The company's work over the past few years centers…

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These Nonprofits Just Shelled Out $5.2 Million to Fund An Experimental Parkinson’s Drug
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These Nonprofits Just Shelled Out $5.2 Million to Fund An Experimental Parkinson’s Drug

According to a story from PR Newswire, the biotech company Mission Therapeutics was just awarded $5.2 million in funding to support MTX325, the company's investigational Parkinson's disease therapy. Intended to…

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Toripalimab Combined with Bevacizumab Meets Primary Endpoint for Advanced Hepatocellular Carcinoma
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Toripalimab Combined with Bevacizumab Meets Primary Endpoint for Advanced Hepatocellular Carcinoma

Shanghai's Junshi Biosciences Co. (The Company) recently announced in Biospace, that its product, toripalimab (TUOYI®), combined with bevacizumab as a first-line therapy for hepatocellular carcinoma (HCC), met its endpoints in a…

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Rare Community Profiles: Volv’s Innovative Approach Harnesses the Power of AI for Rare Disease Detection
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Rare Community Profiles: Volv’s Innovative Approach Harnesses the Power of AI for Rare Disease Detection

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

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SLEEP 2024: Presented Data Highlighted Possible Benefits of ALKS 2680 for Narcolepsy Type 1
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SLEEP 2024: Presented Data Highlighted Possible Benefits of ALKS 2680 for Narcolepsy Type 1

The American Academy of Sleep Medicine and the Sleep Research Society held the 38th annual SLEEP meeting in early June 2024. The meeting allows stakeholders across the sleep sphere to…

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