When Camille Wahl was a child, she loved Irish step dance. She translated her love physically, becoming a competitive dancer until she was ten years old. It was then, reports…
Even with available therapies, pancreatic cancer is incredibly tough to treat and comes with a poor prognosis. Only 12% of people diagnosed with pancreatic cancer are still alive after…
A Phase 2 clinical study set out to determine whether prexigebersen, alongside decitabine and venetoclax, would be effective in treating and stopping the progression of a rare cancer of…
If gastric cancer is localized (has not yet spread), it carries a significantly higher 5-year survival rate of 72-75%. But once the cancer spreads, the rate falls lower: 35%…
Having a rare or uncommon condition can be undoubtedly overwhelming. The diagnosis comes with associated mental health, financial, physical, and social burdens that can weigh heavy on your mind. But…
According to a story from Market Screener, the drug company AbbVie recently announced that its investigational therapy epcoritamab has received a positive opinion from the European Union's Committee for Medicinal…
According to a story from WGN-TV Chicago, treatment for esophageal cancer can be grueling on patients, even if successful. Surgery is often a critical aspect of the process and can…
According to a story from news-medical.net, a recent study found that elevated body-mass index (BMI) was associated with worse outcomes for people living with acute lymphoblastic leukemia, with this study…
According to a July 11 article from Maaisha Osman, commercial-stage biotechnology company ADC Therapeutics has voluntarily halted enrollment for the Phase 2 LOTIS-9 study evaluating Zynlonta (loncastuximab tesirine-lpyl) and…
Barrett's esophagus is a condition in which the esophagus, or the tube that connects the mouth to the stomach, becomes damaged, typically due to long-term exposure to stomach acid; this…
Opdivo (nivolumab) is an immune checkpoint inhibitor that binds to PD-1 and contributes to cancer cell death. It has shown efficacy in treating a number of cancer types, both…
In the past, doctors have noticed a trend in hepatocellular carcinoma (HCC). More specifically, research has shown that HCC can grow resistant to certain drugs. This makes it more…
The American Society for Clinical Oncology (ASCO) held its Annual Meeting in late May and early June to discuss clinical guidelines, best practices, and research advancements within the oncology…
The Orphan Drug Act of 1983 was implemented after the U.S. Food and Drug Administration observed drug development issues in the rare disease space. Rare diseases often have smaller…
For the first year and a half of her life, Audrey seemed to be a healthy child. Her parents Tenneil and Tom had no real concerns about her health. So…
Orphan Drug designation is granted to therapies that are designed to treat, diagnose, or prevent rare diseases or conditions. In the United States, a rare condition is one affecting fewer…
Nobody expects their child to be diagnosed with cancer. The idea of facing cancer at a young age can be shocking, terrifying. So when 4-year-old Ash Timmerman seemed slightly "off,"…
Since 2021, biotechnology company Foghorn Therapeutics ("Foghorn") has been developing its therapeutic candidate FHD-286 for a number of malignancies. In one Phase 1 study, researchers are testing the pharmacokinetics,…
It is important to improve the treatment landscape for various types of cancer. Currently, there is a wealth of research searching for cancer cures. Until that happens, though, we…
The Orphan Drug Act of 1983 transformed the rare disease treatment landscape. Prior to the Act’s passage, there was little commercial investment in drug development targeted towards rare conditions.…
“I never thought that it could happen to me.” Unfortunately, we tend to repeat this refrain when facing unexpected hardships in our life from trauma to illness. For many people,…
BCMA is a protein that is found in multiple myeloma cancer cells. CAR-T cell therapies such as Carvytki are engineered to home in on BCMA proteins. According to a…
When someone is diagnosed with a rare disease, cancer, or really any health-related issues, they tend to question what comes next. How will this condition impact them? What is…
Nobody expects to hear that their child, especially their baby, has cancer. But that is just what Chris and Carney Miller were faced with in 2023. The Miller family…
Throughout the world, it can be difficult to advance research and drug development in the rare disease space. In part, this is because many rare diseases have small population…
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