GACI/ARHR2 Awareness Day is February 1st: Spreading rare Disease Awareness
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GACI/ARHR2 Awareness Day is February 1st: Spreading rare Disease Awareness

Generalized arterial calcification of infancy (GACI) and autosomal recessive hypophosphatemia rickets type 2 (ARHR2) Awareness Day is recognized each year on Feb 1. This is a time for spreading awareness…

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Study of the Week: A new Approach to Treat Mitochondrial Disease
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Study of the Week: A new Approach to Treat 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…

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Machine Learning Helps Identify Patterns of Progression in ALS
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Machine Learning Helps Identify Patterns of Progression in ALS

According to a story from myscience.org, neurodegenerative diseases, such as amyotrophic lateral sclerosis, are diseases that progressively worsen over time. However, progression in this disease is not consistent across all…

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Study: One Wet Age-Related Macular Degeneration Drug Appears Superior to the Other
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Study: One Wet Age-Related Macular Degeneration Drug Appears Superior to the Other

According to a story from hopkinsmedicine.org, a study evaluating data from 106 patients living with wet age-related macular degeneration found a significant disparity in outcomes when comparing patients that received…

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FY23 Appropriations Act Includes Increased Funding and Provisions for the Rare Disease Community
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FY23 Appropriations Act Includes Increased Funding and Provisions for the Rare Disease Community

On December 23, 2022, President Joe Biden officially signed the Consolidated Appropriations Act, 2023. This bill sets in stone the federal government's budget for the year, so naturally it includes…

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FDA Clears IND Application and Grants Orphan Drug Designation to an Experimental Therapy for Progressive Multifocal Leukoencephalopathy
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FDA Clears IND Application and Grants Orphan Drug Designation to an Experimental Therapy for Progressive Multifocal Leukoencephalopathy

According to a story from PR Newswire, the cell therapy company Cellevolve Bio, Inc., recently announced that the US Food and Drug Administration (FDA) has advanced the development of its…

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BTK Inhibitors for Waldenström’s Macroglobulinemia: Comparisons, Benefits, and the Future
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BTK Inhibitors for Waldenström’s Macroglobulinemia: Comparisons, Benefits, and the Future

According to a story from AJMC, a recent critical review evaluated the class of therapies known as Bruton tyrosine kinase (BTK) inhibitors and their use in the treatment of Waldenström's…

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Primary Hyperoxaluria Drug’s Label Receives Expansion to Include Patients with More Severe Disease
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Primary Hyperoxaluria Drug’s Label Receives Expansion to Include Patients with More Severe Disease

According to a press release published on globalgenes.org, the US Food and Drug Administration (FDA) has expanded the label for Oxlumo, a drug developed by Alnylam Pharmaceuticals as a treatment…

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