Nearly all cases of Wilms tumors, a rare form of pediatric kidney cancer, are diagnosed before a child turns 10 years old. When Maddie Newburn was first diagnosed, she was…
Drug development is important in creating new therapeutic options for patients with rare or chronic illnesses. One incentivizing program is the Orphan Drug program. Orphan Drug designation is granted to…
One key facet of the healthcare system is working to improve patient outcomes and finding new, effective, and innovative lines of treatment. According to Hematology Advisor, a study showed that…
On August 11, 2021, biopharmaceutical company Lantern Pharma, Inc. ("Lantern") shared the news that its small molecule drug candidate, LP-184 (hydroxyureamethylacylfulvene), received Orphan Drug designation from the FDA. This status…
Unfortunately, recurrent ovarian cancer can be difficult to treat and often comes with a poor prognosis. However, according to a news release from biopharmaceutical company IMV Inc. ("IMV"), the company…
Typically, statins, or HMG-CoA reductase inhibitors, are prescribed to help reduce cholesterol levels in the blood. But could this class of drugs be repurposed for the treatment of another group…
The FDA has recently granted ONCOFID®-P the Orphan Drug designation for the treatment of malignant mesothelioma (MM). This designation is reserved for therapies indicated for rare diseases, and it provides…
Orphan drug designation is granted in the United States to expedite the development and review of drugs and biologics intended to treat patients with rare conditions (over 200,000 Americans). Once…
HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma patients haven't seen a drug approval for their disease state in a decade. That is until the FDA approved Keytruda in combination with…
The FDA has recently granted Orphan Drug designation to TST1001, Transcenta Holding Limited's monoclonal antibody for gastric cancer/gastroesophageal junction (GC/GEJ). Prior research has demonstrated TST1001's anti-tumor activity, and ongoing research…
In the past, ibrutinib has been used to treat patients with a variety of conditions, including chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), and chronic graft-versus-host disease (cGVHD). According…
Clinical guidelines are often used to improve patient treatment. These guidelines use evidence reviews, as well as an examination of other standards-of-care, to optimize patient care and outcomes. According…
If I told you that tiny bubbles could be used to more effectively treat cancer, would you believe me? Well, in the case of neuroblastoma, that might be correct. According…
On July 28, 2021, biopharmaceutical company Thetis Pharmaceuticals ("Thetis") shared that its small molecule Resolvin E1 (RvE1) therapy, TP-317, received Orphan Drug designation from the FDA. While TP-317 has…
According to a July 27 news release from biopharmaceutical company Day One Biopharmaceuticals ("Day One"), the company's therapeutic option DAY101 received Rare Pediatric Disease designation within the United States. Altogether,…
The FDA has recently approved KEYTRUDA (pembrolizumab) in combination with LENVIMA (lenvatinib) for the treatment of advanced endometrial carcinoma that is not mismatch repair deficiency or microsatellite instability-high. This approval…
The symptoms of terminal pancreatic cancer are subtle. The disease is usually not diagnosed until it has substantially progressed, leaving a dismal five-year overall survival of five to ten percent.…
In the past, researchers have shown that acute myeloid leukemia (AML) may be driven by certain genetic mutations. For example, some mutations might cause the abnormal or excess production of…
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…
In the United States, the Orphan Drug Act was developed to expedite the development and review of drugs or biologics intended to treat rare or life-threatening conditions. For the purpose…
In a video conversation recorded for Cancer Network, Kristie L. Kahl and Javier L. Munoz, MD, MS, FACP discussed treatment for relapsing/remitting follicular lymphoma and the importance of the sequencing…
The healthcare system is designed to provide safe, effective, and beneficial care for all. Unfortunately, the current system represents a wealth of socioeconomic and racial disparities. Past studies have shown…
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 the Cancer Network, orelabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, was recently granted Breakthrough Therapy designation within the United States. While the treatment is also indicated for patients…
KRYPOLIS (carfilzomib) recently received conditional approval in China for the treatment of relapsed/refractory (R/R) multiple myeloma. Licensed by BeiGene, this drug is indicated for adults with R/R multiple myeloma who…
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