In the United States, during the drug development and approval process, drug developers are able to request Fast Track designation. This process is designed to facilitate and expedite the…
Medical research and clinical trials are extremely important tools in learning more about specific diseases, as well as understanding and evaluating potential treatments. For example, the Phase 2 VIBRANT…
According to a story from Healio, a recent phase 2 clinical trial found that treatment with vinorelbine plus active supportive care offered greater progression-free survival than active supportive care on…
At the end of June 2022, biopharmaceutical company InflaRx N.V. (“InflaRx”) shared via press release that its product vilobelimab recently earned Orphan Drug designation in both the United States…
According to a recent article, a study has shown that keratoconus patients who do not get their timely corneal crosslinking will have permanent changes in their corneal structure. Keratoconus…
The history of mesothelioma dates back to 1767 as being the earliest record of tumors found on the lining of the lungs. In 1909 J.G. Adami introduced the term…
According to a story from Charcot-Marie-Tooth News, a recent study using a mouse model of Charcot-Marie-Tooth disease type 1A produced some interesting results. The researchers found that administration of theophylline…
Findings from the MITO23 Trial investigating the outcome of the chemotherapy drug trabectedin against physician’s choice to treat BRCA-mutated patients with ovarian cancer were presented at ASCO’s 2022 Annual…
Individuals with Duchenne muscular dystrophy have mutations in the gene that makes the protein dystrophin. This gene is the largest gene in the human body, with 79 exons. When there…
According to a story from Globe Newswire, olipudase alfa (marketed as Xenpozyme) has been approved by the European Commission for the treatment of non-CNS related symptoms of acid sphingomyelinase deficiency…
In a press release on the company website, biopharmaceutical company AbbVie shared that preliminary data was available regarding navitoclax, an investigational therapy, for individuals with primary myelofibrosis. The data…
The ENDO 2022 Annual Congress took place from June 11-14, 2022. During the Congress, stakeholders met to discuss care, treatments, and research within endocrinology. In a news release from…
The most common gastrointestinal tumors in the world are esophageal cancers. A recent article in DovePress reports that the malignancy is also the eighth most common worldwide. In China…
According to a recent article, the PI3K inhibitor umbralisib used to treat marginal zone lymphoma has had its approval withdrawn by the FDA. Marginal Zone Lymphoma (MZL) Marginal…
In many cases, orally administered PDE5 inhibitors – such as Cialis, Levitra, and Stendra – have been used to treat erectile dysfunction. These vasodilating drugs were found to be…
The American Society of Clinical Oncology (ASCO) Annual Meeting (“ASCO 22”) took place this year from June 2nd through the 6th, 2022. During this Annual Meeting, stakeholders within the…
The study was intended to recruit 6,000 Alzheimer's patients, but even after recruiting had proceeded for seven months, just 29 patients were enrolled. As a result, the trial was cancelled,…
The novel program called BeginNGS™ was described in a recent article which has a goal of testing for five hundred disorders and several thousand infants. Rady Children's Institute for…
According to a story from Globe Newswire, the global healthcare company Novo Nordisk recently announced encouraging findings from its phase 3 clinical trial. This study was evaluating somapacitan (marketed as…
Unfortunately, it can sometimes be difficult to get companies to perform research and develop therapeutics for rare diseases, but the FDA came up with a process to spur research…
Clinical trials are useful not only for initially establishing the efficacy of treatments for rare diseases, but for demonstrating that these results are durable as well. The MPOWER trial, which…
In normal development, genes are usually inherited in pairs - with one copy from the father and one inherited from the mother. When one copy of the TCF4 gene, which…
According to a story from news-medical.net, the International Rett Syndrome Foundation (IRSF) has announced the launch of its Clinical Trial Committee. The committee was implemented with the goal of helping…
In the United States, a rare disease or condition is one which affects fewer than 200,000 individuals. Alternately, a rare condition in the European Union (EU) is considered one which…
Pancreatic cancer is one of the deadliest forms of cancer. According to the American Cancer Society (ACS), only about 11% of patients are expected to survive more than five years…
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