Currently, there are no FDA-approved treatments for nonalcoholic fatty liver disease (NAFLD) or its more advanced version: nonalcoholic steatohepatitis (NASH). This is partly due to the lack of understanding surrounding…
Every game, the men of Northwestern Football leave the tunnel to a sign with an important reminder: “trust yourself.” Each player touches that sign before they head to the field,…
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…
Currently, about 90% of cystic fibrosis (CF) patients have a treatment option. This percentage jumped from 50% to 90% upon the approval of Trikafta. However, the remaining 10% of CF…
Every year, there are numerous unique ways to get involved in the rare disease community and help raise awareness. One example? The Harvest Hope for PWS event -- a virtual…
BRUKINSA (zanubrutinib), an orally administered Bruton's tyrosine kinase (BTK) inhibitor, has been approved in various countries for the treatment of mantle cell lymphoma (MCL), Waldenström's macroglobulinemia, and marginal zone lymphoma. For…
According to a story from express.co.uk, Crohn's disease is the more common form of inflammatory bowel disease; therefore, it is typically associated with symptoms that affect the digestive system and…
According to a story from echolive.ie, ten year old Adam Terry from Cork, Ireland, has directly appealed to Micheal Martin, the Taoiseach (or prime minister) of the country in order…
Rare Diseases South Africa (RDSA) and The Rare Diseases Access Initiative (RDAI) have recently come together to talk about the issues that those living with rare diseases in South Africa…
A new ale is hitting Doubleday’s and The Bit in Dobbs Ferry with a very special purpose. Named "Jacob the Warrior" IPA, this beer was created to raise awareness for…
Rats are a part of New York City - if you've ever been, you've definitely seen them. Most of the time, they're just pests on the streets or subway that…
According to a news release from biotechnology company LAVA Therapeutics N.V. ("LAVA"), the company's therapeutic candidate LAVA-051 recently earned Orphan Drug designation from the FDA. The therapy is designed for…
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 news release from biopharmaceutical company Mereo BioPharma Group plc ("Mereo"), the Osteogenesis Imperfecta Foundation (OIF), and the Osteogenesis Imperfecta Federation Europe (OIFE), enrollment is now complete for…
Maya Doyle is a health social worker specifically focused in pediatrics and the transition from pediatric care to adult care. She has been practicing for twenty years. Now, she works…
Currently, there are no known cures for CDKL5 deficiency disorder, a rare genetic disorder characterized by seizures and severe developmental delays. Treatment, rather, is symptomatic and supportive. However, a news…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
A new partnership has been formed between Global Genes and the Rare Disease Diversity Coalition (RDDC) - created by the Black Women's Health Imperative - in an effort to increase…
In the past, some researchers believed that fibromuscular dysplasia was a rare disease. While some researchers estimate that the incidence is actually much higher than reported, there is still a…
A lack of awareness is a problem that many rare diseases face. Small patient populations, limited research, and other factors all play a role in this issue. Luckily, there are…
Joey Renick was 3 years old when he first received a diagnosis of acute lymphoblastic leukemia (ALL). The Missouri native went through three years of treatment before going into remission…
The very first National Census of Patients with Rare Diseases in Mexico began October first of 2021 by the General Health Council (CSG). This census will last until January 15th…
The National Organization for Rare Disorders (NORD) held its virtual Rare Diseases and Orphan Products Breakthrough Summit on October 18-19, 2021. Patient Worthy was able to sit in on some…
Atara Therapeutics has released data from its Phase 1 open-label extension (OLE) trial of ATA188, a treatment for progressive multiple sclerosis (MS). This allogeneic T-cell immunotherapy targets B cells and…
Previously, clinical-stage genetic medicines company Homology Medicines, Inc. ("Homology") submitted an Investigational New Drug (IND) application to the FDA for HMI-103, an investigational gene editing therapy designed to treat phenylketonuria…
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