For as long as she can remember, Tiffany Williams wanted to make a difference, especially in the healthcare field. She earned her doctorate degree and began working as a…
In the past, it was difficult to incentivize the development of treatments for rare diseases, given issues with both profitability and small patient populations. However, all people are deserving…
Known as “Slydah,” Chicago White Sox relief pitcher (RP) Liam Hendriks has done amazing things on the field. In September/October 2021, Hendriks was named the American League Reliever of…
For many people living with mantle cell lymphoma (MCL), the standards-of-care include Bruton's tyrosine kinase (BTK) inhibitors. But there is a subset of patients who do not respond well,…
Friends and family refer to 41-year-old Teddy Higgins as kind, selfless, and loving. Given all of his support for others, his friends and family are now working to support Teddy…
When Leonidas Boyne was born, he seemed—for all intents and purposes—to be a healthy, happy child. At five months old, he is still a bubbly, happy baby with a smile…
High-risk neuroblastoma can be difficult to treat in children. Some research suggests that between 40-50% of children with high-risk neuroblastoma relapse following treatment, causing poor outcomes. When Flora Gentleman was…
In the United States, the Food and Drug Administration (FDA) grants Orphan Drug designation to drugs or biologics which intend to treat, diagnose, or prevent rare conditions. “Rare” conditions are…
An antidepressant drug that was approved by the FDA in 1959 is being put to new and promising use to fight hard-to-treat breast cancers. The antidepressant, imipramine, was approved to…
In 2021, the U.S. Food and Drug Administration (FDA) approved zanubrutinib (Brukinsa) for adult patients with relapsed or refractory (R/R) marginal zone lymphoma. At the 64th Annual American Society for…
In the United States, Fast Track is the process designed to facilitate the development, and speed up the review, of therapies designed to fill an unmet need or treat…
Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat, diagnose, or prevent a rare disease or condition. “Rare,” in the United States, is…
While alveolar soft part sarcoma (ASPS) is considered to be relatively slow-growing, it is necessary to carefully manage this rare soft tissue sarcoma to avoid metastasizing. Surgery is one…
Continued From Part One Multiple myeloma is caused by the buildup of abnormal white blood cells that form tumors found primarily in bones. Available treatments work for short periods…
Unfortunately, the prognosis associated with glioblastoma is poor; some studies suggest that survival rates are 40% within one year of diagnosis and 17% within two years, with less than 7%…
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…
Med City News published an article by Bruce Feinberg, M.D. at Cardinal Heath, examining CAR-T treatments and the impact they have had in medicine during the past five years. CAR-T's…
Rare diseases are, by their nature, rare—and because of this, it can be difficult to spur drug development. The FDA worked to overcome this through the creation of the…
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…
Researchers at Brown University, in collaboration with an MIT research team, discovered a method of penetrating the blood-brain barrier (BBB) in mice and delivering medication to treat lethal tumors…
The CDC lists lung cancer as the third most common type of cancer in the United States with the highest death rate. Estimates are given of 2.2 million patients worldwide…
Diffuse midline glioma (DMG) refers to a rare and extremely aggressive brain tumor that is most common in children. Chemotherapy and radiation are the current standards-of-care. Unfortunately, even with…
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…
Mahesh Karande, the President and CEO of Omega Therapeutics, is incredibly passionate about epigenetics. And it shows: in the work that he and his team do, in the therapies they…
So far, the Phase 2 EMPOWER-CSCC-1 clinical study has evaluated cemiplimab (also known as Libtayo) in individuals with locally advanced or metastatic cutaneous squamous cell carcinoma (cSCC). Data from…
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