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…
According to a story from ca.sports.yahoo.com, the biotechnology company Galecto, Inc. has announced encouraging findings from an intermediate assessment of its phase 2a clinical trial. This trial is evaluating the…
According to a story from empr.com, the Oncologic Drugs Advisory Committee, part of the US Food and Drug Administration (FDA), recently voted 16 to 0 against the investigational drug omburtamab…
According to a story from PR Newswire, the biopharmaceutical company AMO Pharma Limited has recently announced the completion of enrollment for its REACH-CDM clinical trial. This trial will be investigating…
A new awareness campaign called RKD & Me has recently kicked off with the goal of spreading awareness of rare kidney disease (RKD). The project is a collaboration between the…
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…
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…
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 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…
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 PR Newswire, the pharmaceutical company Saol Therapeutics has recently announced that it has completed its enrollment goals for its phase 3 clinical trial. This trial…
Parents and doctors were concerned about Chuck Goodman's health from an early age. In fact, they thought he had a deadly case of childhood leukemia. At around age five, Chuck…
On December 8, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
According to a story from finance.yahoo.com, a new treatment regimen has been approved by the US Food and Drug Administration (FDA) for hepatocellular carcinoma that cannot be treated with surgery.…
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…
According to a story from morningstar.com, the pharmaceutical company Horizon Therapeutics plc recently announced the completion of enrollment for its phase 3 clinical trial in Japan. This trial is testing…
According to a story from CNN, a new treatment was recently approved by the US Food and Drug Administration (FDA) for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's…
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 PR Newswire, Immunic, Inc., a biopharmaceutical company prioritizing the development of oral immunology medicines for the treatment of chronic inflammatory and autoimmune disorders, has reported…
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 press release from the US Food and Drug Administration (FDA), the agency recently approved a new treatment for multiple myeloma, a type of rare blood cancer. The…
In recent years, treatment options for cystic fibrosis, a rare disease, have increased significantly in number and in effectiveness. However, many of the most effective treatments, developed by Vertex Pharmaceuticals,…
According to a story from mirror.co.uk, Emma Timofte, age six, was diagnosed with a rare disease called incontinentia pigmenti. The diagnosis came when she was only a few weeks old.…
There is a certain time of year in which the trees change color, the air becomes crisp and we pour gravy on everything. As I am typing this, I am…
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