A Tyler, Tx artist, mother, wife, and friend has her community rallying behind her in her fight against oligodendroglioma, a primary central nervous system cancer. According to the Tyler Morning…
Orphan Drug designation in the United States is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions, defined as those affecting under 200,000 Americans.…
Years ago, talabostat (BXCL701) failed during a Phase 3 clinical trial to evaluate the therapy for patients with pancreatic cancer. However, drug developer BioXcel Therapeutics (“BioXcel”) did not give up…
According to a November 3, 2021 news release from biopharmaceutical company OS Therapies, the company’s therapeutic candidate OST-HER2 (OST31-164 / Listeria monocytogenes) received Rare Pediatric Disease Designation from the FDA.…
In 2019, Mike Smith, a father of two, was initially diagnosed with ocular melanoma in his right eye. Shortly after, Mike made the necessary - but difficult - decision to…
According to a press release from Ikena Oncology, the FDA has recently accepted an Investigational New Drug (IND) application for Ikena's investigational drug candidate, IK-930. This TEAD inhibitor is intended…
Sometimes, treatments used in conjunction with each other can confer benefits for patients. According to the Cancer Network, for example, treating platinum-resistant ovarian cancer using nemvaleukin alfa and pembrolizumab (Keytruda)…
Currently, a glioblastoma multiforme diagnosis comes with a relatively poor prognosis: 25% survival after 1 year, and 5% after 5 years. Researchers have been looking to not only expand their…
When drug developers receive marketing approval in different countries, they expand medication access and allow patients across the globe to have better treatment. According to Korea Biomedical Review, Korea recently…
BRUKINSA (zanubrutinib), an orally administered Bruton's tyrosine kinase (BTK) inhibitor, has been approved in various countries for the treatment of mantle cell lymphoma (MCL), Waldenström's macroglobulinemia, and marginal zone lymphoma. For…
According to a news release from biotechnology company LAVA Therapeutics N.V. ("LAVA"), the company's therapeutic candidate LAVA-051 recently earned Orphan Drug designation from the FDA. The therapy is designed for…
In a Case-Based Roundtable for Targeted Oncology, Dr. Haris Ali, an associate professor with the City of Hope Department of Hematology and Hematopoietic Stem Cell Transplantation, discussed a case of…
On September 30, 2021, the National Organization for Rare Disorders (NORD) hosted an educational webinar titled "Rare Cancers: Breaking Down Barriers to Diagnosis, Treatment, and Research." The webinar focused on…
To best address the needs of patients with rare diseases and rare cancers, additional treatment options are necessary. But developing these treatment options can be time-consuming and sometimes difficult.…
Screening tests are used to identify potential signs related to certain diseases or conditions. For example, newborn screening, performed at birth, gives insight into whether or not a child has…
According to Healio, the FDA recently granted Orphan Drug designation to nadunolimab (CAN04), a treatment candidate for patients with pancreatic cancer. Orphan Drug designation is given to drugs or biologics…
A new collaboration between the University of Texas's MD Anderson Cancer Center and the Rare Cancer Research Foundation aims to solve a problem that has been a major obstacle for…
In the past, caffeine has been somewhat of a complex or even controversial topic. While some research shares that it is bad for health, others laud caffeine’s ability to enhance…
Clinical trials have many endpoints - or milestones they are looking to achieve. These endpoints help signify the safety, efficacy, and tolerability of the treatments. In the Phase 3…
September will soon come to a close, but did you know that this month is also Ovarian Cancer Awareness Month? According to a story from MSN, Michelle Barrenechea, an EMT…
Funding is a problem that many rare diseases face when it comes to research. Cholangiocarcinoma, also known as bile duct cancer, is no exception. Because of this, it is often…
In the past, Exelixis' drug CABOMETYX (cabozantinib) has been approved for use in a variety of patients. For example, the treatment is approved for patients with renal cell carcinoma (RCC)…
According to a press release from Business Wire, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently issued a positive opinion in regards to the…
Steven Rodriguez was nineteen years old attending college in California when he began to have severe headaches. A recent article from the UCSF Science Center gives an account of Steven’s…
According to a story from Targeted Oncology, the US Food and Drug Administration has given Orphan Drug designation to ARX788, an experimental therapy. The designation was given for the treatment…
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