Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to a story from MENAFN, the Delhi High Court recently issued a directive to the National Consortium for Research, Development, and Therapeutics for Rare Diseases to convene and move…
According to a story on msn.com, Alijah Cunningham is living with the rare disease Apert syndrome, resulting in the bones of his toes and fingers fusing together. Now three years…
On March 9-10, 2023, Dynamic Global Events hosted the virtual Global Innovation in Patient Advocacy conference. This event was geared towards various stakeholders in the rare disease space and was…
Jamas and Margot LaFreniere started the Sophie's Hope Foundation in 2020 shortly after Sophie, their daughter, was diagnosed with a rare disease: glycogen storage disease type 1B (GSD1B). The mission…
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…
March 24 is being recognized this year as Lipoprotein(a) Awareness Day, a time to spread awareness among the general public and the medical field about elevated lipoprotein(a), a condition which…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to a story from people.com, beverage entrepreneur and former star on Real Housewives of New York City Bethenny Frankel has taken to the social media app Instagram to share…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
According to a story from sciencex.com, Queen's University Belfast, University College Dublin, and a team of 33 other partners have come together to start the All-Ireland Rare Disease Interdisciplinary Research…
According to a story from koin.com, graft-versus-host disease is a condition that can affect recipients of stem cell or organ transplants. Dr. Corey Cutler, Medical Director, Stem Cell Transplant Program,…
According to a story on pharmaphorum.com, the UK's National Institute for Health and Care Excellence (NICE) has given a positive opinion for the drug vutrisiran (marketed as Amvuttra) as a…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to a story from World Pharma Today, Andelyn Biosciences, Inc., which is a gene therapy Contract Development and Manufacturing Organization (CDMO), has recently struck a partnership with Odylia Therapeutics,…
According to a story from VICE News, Congressman George Santos was recently elected to Congress in the 2022 mid-term elections. Since then, his past has been placed under scrutiny, and…
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…
According to a story from Globe Newswire, the gene therapy company uniQure recently announced that it has received conditional marketing authorization for its gene therapy etranacogene dezaparvovec (marketed as HEMGENIX®).…
From February 28 to March 2, rare disease advocates met in Washington, DC for the 12th Annual Rare Disease Week on Capitol Hill. Organized by Rare Disease Legislative Advocates (RDLA),…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
February 28th is recognized as Rare Disease Day around the world each year. This day is intended to bring recognition and awareness to the plight of the millions of people…
The Foundation for Angelman Syndrome Therapeutics (FAST) recently entered a partnership with Rush University in order to establish a clinical trial and translational research center for rare neurodevelopmental disorders.…
When Richard Poulin's daughter Rylae-Ann was born, it wasn't long before he and his wife began to wonder if something was wrong. They both felt confident in understanding the…
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…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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