Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
by Danielle Bradshaw from In The Cloud Copy Bone grafts, although very helpful, can result in hard to treat infections and typically result in the patient needing to be put…
Medical professionals are looking towards patient-reported outcomes more and more as a source for research. In fact, a study titled "Patient reported impact of Charcot-Marie-Tooth disease: protocol for a real…
United Therapeutics Corporation has recently presented data from the Phase 3 INCREASE trial, which evaluated Tyvaso as a treatment for pulmonary hypertension associated with interstitial lung disease. Results were presented…
According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access…
According to a story from UT Health San Antonio, a team of researchers have discovered in a new study the mechanism behind the activation of magnesium ions in cells. The…
A recent study published in the Journal of Translational Medicine describes an accurate and objective approach that aims to evaluate the severity of myalgic encephalomyelitis, also known as chronic fatigue syndrome. This…
According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…
According to a recent article in ScienceAlert, scientists have conducted experiments in mice activating a protein called protrudin that gives damaged nerve fibers the ability to regenerate. Currently, once nerve…
In a recent press release, biopharmaceutical company Rallybio announced that the first participant was dosed in a Phase 1/2 clinical trial. Currently, only healthy male participants are enrolled in the…
The European Medicines Agency has accepted Biogen's Marketing Authorization Application (MAA) for review. The application is for aducanumab, a treatment for Alzheimer's disease. As this condition faces an unmet medical…
While clinical trials can be extremely helpful in the development of new treatments, not all trials go as planned. Recently, biotechnology company Regeneron Pharmaceuticals ("Regeneron") discovered this firsthand. As the…
Pancreatic cancer is a rare but severe cancer, and it is often difficult to diagnose until it has advanced. When you combine this with a lack of knowledge and awareness…
Tuberous sclerosis complex (TSC) is a rare disease that, like many other rare diseases, is not yet fully understood by medical professionals. Because of this, it is very difficult to…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Bryan and Bradford Manning recently gave an interview to the Associated Press. The brothers told the story of their vision loss as a result of a rare degenerative eye disease.…
According to a story from PR Newswire, a new collaboration between the Hairy Cell Leukemia Foundation (HCLF) and the Leukemia & Lymphoma Society (LLS) will aim to support critical research…
As reported in CBS News, sisters Izzy and Ailbhe Keane were graced to be born with a natural partner in crime. Their creative flair and eye for style came naturally.…
According to a story from Charcot-Marie-Tooth News, CMT UK, a United Kingdom-based nonprofit focused on Charcot-Marie-Tooth disease, has released a series of seven exercise videos tailored to patients as part…
According to a story from fiercepharma.com, the US Food and Drug Administration (FDA) was willing to approve the drug Tibsovo as a treatment for acute myeloid leukemia (AML) following encouraging…
According to a story from SELF, Jaime Stathis lives with a rare condition called misophonia, in which certain trigger sounds can cause negative emotional states and outbursts. Jaime discusses how…
In a press release from Sanofi, a global biopharmaceutical company, the company's experimental therapy candidate olipudase alfa has demonstrated the ability to substantially improve spleen volume and lung function in…
Colorectal Cancer (CRC) is now the third leading cause of cancer-related deaths in the U.S. A report in Foundation Medicine highlights data on the increase in the number of cases…
The FDA has recently approved of an updated label for Endari, a treatment for sickle cell disease (SCD). This new label will give medical professionals better information, allowing them to…
According to GlobeNewswire, the results from a Phase 1 study of lirentelimab have been released. Allakos developed this medication as a treatment for eosinophilic and mast-cell related diseases. In this…
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