Recent headlines in research and advocacy show promising news for the treatment of rare diseases.
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The National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1 and Sandhoff diseases, is hosting the first-ever, ExternallyLed Patient-Focused Drug Development Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on Thursday, February 15, 2024.
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Edgewise’s investigational drug is a pioneering treatment for Duchenne and Becker muscular dystrophies.
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Know Rare's Medical Advisor, led the Mass General for Children’s Storybook Ball, raising $1.9 million for innovative healthcare and research, while sharing her own personal rare disease journey, and highlighting Know Rare's mission to connect and empower individuals living with rare conditions.
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A new treatment for myasthenia gravis, a rare neuromuscular condition, has been approved by the FDA thanks in part to the success of a clinical trial that Know Rare helped to recruit patients for.
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In a 2021 report, authors Ahmad Saud, R Naveen, Rohit Aggarwal & Latika Gupta, all well-known experts in myositis, discuss recent findings about the relationship between COVID-19 and myositis.
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Discover how forward-thinking researchers are designing clinical trials to accommodate people’s lives and needs, helping with clinical study participation and the development of much-needed treatments.
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A recent study, involving children of ages 12 and older with PA or MMA, found an interesting approach in assessing individuals’ abilities to understand, think, and reason.
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At times, managing a rare condition can feel like an isolating reality. We’ve rounded up notable names who break with that status quo, bringing rare conditions—and the stories behind them—into the spotlight.
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Learn about a terrible story that occurred in 1989, when a woman named Patricia Stallings was wrongly convicted for the death of her son.
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Management of chronic kidney disease is complicated by many things, such as fluid retention, anemia, and effects on multiple organs in the body. In India, where treatments can be too expensive and beyond the reach of the majority of the population, many have relied upon yoga as an alternative therapy.
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Rare advocates, such as Nadia Bodkin, are shining a light on racial and ethnic inequities in healthcare systems. Learn how Rare Advocacy Movement (RAM), co-founded by Bodkin, and the rare disease community are fighting to make care more equitable for all.
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A recent New York Times article discusses the inexactness of prenatal testing and how false positives about genetic markers of rare disease can cause anxiety. Read genetic counselor Elizabeth Kearney’s response, where she expertly addresses the inaccuracies and assumptions made in the article.
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For people with primary mitochondrial myopathies (PMM), it is reassuring to know new studies are continuing to explore activators of PPAR delta as a possible way to stimulate mitochondrial activity, increase energy production, and improve the performance of skeletal muscle cells.
The RDDC was established at a pivotal time, as the United States continues to grapple with the impact of the COVID-19 pandemic and the glaringly evident racial disparities that exist regarding infection rates, treatment, and access to care.
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From trailblazing researchers to game-changing public health advocates, women in medicine historically faced marginalization due to their gender, and, in some cases, their race. Nonetheless, they changed the playbook for the physicians who would follow them, as well as for the patients who would benefit from their discoveries in the future.
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Sponsored by Boehringer Ingelheim
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