LiPlaCis is a formulation of cisplatin that delivers its agent directly to cancerous tumor sites. Its efficacy has so far only been studied within breast cancer patients. However, scientists have reason to…
Actress Marcia Cross, best known for her role as Bree Van de Kamp on Desperate Housewives, has shared that she is now healthy after battling anal cancer in a series…
According to a recent press release from Bluebird Bio, the European Medicines Agency has accepted and will review their marketing authorization application (MAA) for the investigational gene therapy LentiGlobin™ as…
According to a story from MarketWatch, the biopharmaceutical company MediciNova recently announced that the company's product ibudilast was granted Orphan Drug designation by the FDA. The designation was granted for…
If you think you have to be middle-aged or older to check your cholesterol, think again. Laura Livesey is a relatively young blogger (you can check out her blog, Confidence…
A drug called Coversin is being investigated as a potential treatment for four orphan diseases: bullous pemphigoid (BP), atypical keratoconjunctivitis (AKC), thrombotic microangiopathies, and paroxysmal nocturnal hemoglobinuria (PNH). Recently, new…
Happy October, everyone! We hope everyone's enjoying this new ~fall weather~ and the endless supply of pumpkin spice flavored food products. This week, we're highlighting four FDA announcements we think…
PharmAbcine has announced that the United States Food and Drug Administration has responded to the Investigational New Drug application they submitted on behalf of an experimental drug called TTAC-0001, which…
According to a story from 9news.com.au, the first sign that Jemima Leydon had ovarian cancer was a stomachache. Her mother Megan assumed that it was just a stomach bug that…
A new drug designed to treat MAC lung disease as part of a combination antibacterial drug regimen in adults with limited treatment options has been granted Accelerated Approval by the…
Researchers have carried out a literature review to investigate MPS III. After reviewing forty-six papers, they concluded that more research needs to be carried out. You can find the original study…
A diagnosis of Cri du Chat (or 5P-), used to mean institutionalization. The name is french meaning "cry of a cat" which comes from the distinct sound children with the illness…
According to a report by medicalexpress, recent research from a DNA sequencing study revealed new information about Tourette syndrome. Led by researchers at UC San Francisco, this study represents the…
The United States Food and Drug Administration has approved Copiktra™ (duvelisib). The drug is now approved as a treatment for adult patients diagnosed with refractory or relapsed chronic lymphocytic leukaemia…
The company Genkyotex has announced that they have finished enrolling participants for a Phase 2 clinical trial of the investigational drug GKT831 in patients who have primary biliary cholangitis. The…
The clinical research process is a thorough and time consuming method in which new drugs, therapies, and medical devices are rigorously tested before being made available to the public. Through…
Encouraging interim data has been shared from two studies of risdiplam (RG7916), a drug being researched as a potential treatment for spinal muscular atrophy types 1, 2, and 3. For…
According to a story from MedCity News, a recent study revealed a potential weakness of CAR-T cell immunotherapies that reveals the need for precision and caution in the extraction process.…
How it all started Joy Milne is a retired nurse who helped diagnose her husband with Parkinson's disease. 12 years before the official diagnosis, Joy noticed a change in her…
A past, current, and future problem – underrepresentation in clinical trials, particularly for rare diseases. Why does representation matter? Besides, you know, the basics of equality, drugs can react differently…
October is Dysautonomia Awareness Month! Before we get into advocacy, let's touch on basic awareness... What is dysautonomia? Dysautonomia is the umbrella term that encompasses several different conditions that cause a…
Lindsey Sutton showed signs of familial chylomicronemia syndrome as early as five weeks old. Sutton is now 28 years old. Because of her condition, she must adhere to a very…
A Texas company, which opened in 2015, promised a new treatment for a number of conditions. Simply coming in and subscribing to a routine of IV treatments could treat cancer,…
According to a story from wtvbam.com, the drug development company Alnylam Pharmaceuticals has recently stated that it is looking less likely that its gene silencing therapy givosiran will be getting…
Just days after my CVID diagnosis, I was invited to a support dinner. It was at this support dinner that I became aware of how unique my situation was; I…
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