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…
If you’ve been there, then you know. Patients experiencing cancer, especially the rare aggressive types such as pancreatic cancer, and requiring targeted treatment for the disease may soon be…
If you live with any form of pain, I’m hoping the following information will be of help. Many of us look at pain the wrong way that exacerbates our problem,…
The American Society of Clinical Oncology (ASCO) held its Annual Meeting from May 31 - June 4, 2023. During the meeting, attendees discussed a variety of oncological topics, trends,…
Uplifting Athletes, a national nonprofit organization that harnesses the power of sport to support people impacted by rare diseases, celebrated 20 years of Lift for Life with Penn State University…
According to a story from healio.com, an oral medication being evaluated in a phase 2 study was able to increase annualized height velocity in children living with growth hormone deficiency,…
The five-year rate of survival for pancreatic cancer is at 12% having the highest mortality rate of any major cancer. Defence, an aptly-named biopharmaceutical company in Vancouver, B.C. recently…
In January 2023, 23-year-old Angel Anthony Cortez was admitted to the hospital. His health had rapidly deteriorated over the previous days and his family was concerned. Following his admission,…
In May 2023, Pharmaceutical Technology reported that INO-3107, a DNA medicine vaccine candidate developed by biotechnology company Inovio Pharmaceuticals (“Inovio”), received Orphan Drug designation from the European Commission. INO-3107…
June 30th is World Pneumothorax Day and this year we are urging all doctors to ask about family history if a patient presents with a spontaneous pneumothorax. This one…
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…
A recent article published in GlobeNewswire captured the results of the trial that was presented at the 2023 Annual ASCO Meeting from June 2nd to June 6th, 2023 in Chicago.…
At ASCO’s 2023 convention for patients diagnosed with glioma and a specific genetic alteration, Servier Pharmaceuticals announced that vorasidenib, the drug being investigated in its Phase III trial (INDIGO)…
The FDA advisory panel which is composed of specialists who advise the FDA on brain medications, recently voted 6-0 in agreement on that the new drug, Leqembi, was effective…
According to a story from PR Newswire, the QurAlis Corporation, a clinical stage biotech company, recently announced the completion of the review of the Clinical Trial Regulation (CTR) in the…
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…
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023, at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…
When it comes to rare diseases, advancing research is crucial to developing a better understanding of the disease and how it can be treated. However, it can sometimes be difficult…
Zanidatamab is an investigational IgG1 bi-specific monoclonal antibody that targets two non-overlapping HER2 domains. HER2 is overexpressed in a variety of cancer types such as breast cancer and esophageal cancer.…
For as long as he can remember, Eli McCausland has been fascinated by wrestling. His passion for the sport eventually culminated in his co-ownership of Midwest All-Star Wrestling, as well…
TransCode Therapeutics, Inc. (“TransCode”) was founded with a mission to revolutionize the cancer treatment landscape and improve patient outcomes. The company is currently working to uphold this mission through the…
When it comes to the rare disease landscape, there is not enough conversation discussing mental health needs. People undoubtedly need more mental health support and resources during the diagnostic odyssey.…
Sophie Hinchcliffe, best known by her online moniker of Mrs. Hinch, knows how to clean up a mess. After all, her cleaning skills are what launched her into popularity…
Patients who have been heavily pretreated for relapsed/refractory multiple myeloma participated in the phase 2 study of bispecific T cell redirection antibodies. The participants responded to treatment with a combination…
In August 2022, I first interviewed Ryan Sheedy, the Co-Founder of mejo, a company dedicated to creating tools and resources designed to support caregivers and parents. Sheedy discussed how…
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