Remix Therapeutics has achieved a significant regulatory milestone with the U.S. Food and Drug Administration granting Fast Track designation to REM-422, a first-in-class small molecule therapy designed to treat recurrent,…
Rare diseases are challenging. Not just for those affected, but for researchers, physicians, and drug developers. Rare diseases may affect small populations. Many are poorly understood and lack adequate awareness.…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Children born with propionic acidemia require life-long treatment and care; without it, this disorder can lead to coma or even death. While there are supportive treatment options, no current…
Internationally, over 400,000 people die of malaria each year. In many cases, a majority of these deaths occur in sub-Saharan Africa; children are the most vulnerable. However, according to Medical…
Over the last year, news of COVID-19 has dominated the headlines. Caused by SARS-CoV-2, a coronavirus, the global pandemic is now associated with 116 million diagnoses worldwide and 2.57 million…
In late September and early October, the North American Cystic Fibrosis Conference (NACFC) went virtual, as many conferences and symposia this year have been forced to do. However, that does…
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