Prior to 2021, there were no FDA-approved treatments for patients with type 1 plasminogen deficiency, or hypoplasminogenemia. As a result, there was an urgent need to fill this unmet need…
In late May 2021, biopharmaceutical company PTC Therapeutics, Inc. ("PTC") shared that its therapy, PTC923, received Orphan Drug designation within both the United States and Europe. The treatment is designed…
One of the complications of COVID-19 is juvenile dermatomyositis (JDM), a connective tissue disease. In the Children's Medical Center in Iran, researchers documented that from Feb of 2020 to Feb…
In a press release from late May 2021, biotechnology company CancerVAX, Inc. ("CancerVAX"), which works to develop targeted immunotherapy treatments, shared that it had entered into a sponsored research agreement…
From June 5-8, 2021, the European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Congress. Though the event was held virtually, the meeting still expanded…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a June 11 press release from biotechnology company Equillium, Inc. ("Equillium"), positive topline data is now available from the Phase 1b EQUATE clinical trial. Within the clinical trial,…
Gene therapies are becoming an increasingly popular treatment for genetic diseases, especially rare ones. Now, medical professionals are trying a new strategy with gene therapy: adding acetaminophen, also known by…
Currently, the European Hematology Association (EHA) Virtual Congress 2021 is going on from June 9 through 17, 2021. During the Congress, hematologists and other stakeholders will discuss clinical research and…
Currently, the European Hematology Association (EHA) is holding its 2021 Virtual Congress through June 17, 2021. During the event, researchers are expounding on new insights and research within the hematological…
On June 8, 2021, pharmaceutical and biotechnology company Pfizer Inc. ("Pfizer") shared that its Pneumococcal 20-valent Conjugate Vaccine (PREVNAR 20) is now FDA-approved. The vaccine helps protect against 20 different…
The FDA approved Beovu, a treatment for wet age-related macular degeneration (wAMD), back in October of 2019. Now, not even two years later, there are changes that must be made…
According to a post on WAMES (Working for ME in Wales), a recent study used a retrospective analysis to identify potential warning signs for a characteristic symptom of myalgic encephalomyelitis…
The seventy-seven-year-old patient, Mr. S., had lived with chest discomfort for twenty years. Since bypass surgery or stents were not an option, his cardiologist managed to ease his discomfort through…
In a news release from June 2, 2021, biopharmaceutical company Soleno Therapeutics, Inc. ("Soleno") shared an exciting update. The company's investigational treatment, DCCR (diazoxide choline) Extended-Release tablets, received Orphan Drug…
Recent research conducted by Dr, Elif Oral has discovered that leptin therapy may be a viable treatment for nonalcoholic steatohepatitis (NASH), which is a form of fatty liver disease. Dr.…
According to a story from eminetra.com, the month of May was Lyme Disease Awareness Month, which is a suitable time for it as May is often one of the peak…
The gut plays a big role in our bodies, we know that. While some of its roles are obvious, others are still being discovered. In fact, the microbiome of the…
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…
In a study from the University of New Mexico, researchers found the majority of patients with nausea report immediate relief after using cannabis, while 96% of the users reported nausea…
At one point in the past, researchers studied a patient with congenital heart disease (CHD) and came out with a potential cause: SORBS2 gene mutations. However, because the study sample was so…
According to an announcement from the US Food and Drug Administration (FDA), the agency has recently approved a new therapy for Alzheimer's disease. While this is a significant moment for…
In a news release from June 9, 2021, biotechnology company Vertex Pharmaceuticals, Inc. ("Vertex") shared that its therapy TRIKAFTA (elexacaftor/texacaftor/ivacaftor and ivacaftor) was approved for expanded use. Now, the treatment…
According to a story from the Bognor Regis Observer, 25 year old Kieren Rogers had his first stroke last August. It was a minor event, but he still needed to…
Early diagnosis and prompt treatment are absolutely necessary for the best outcomes in many rare disorders. Keratoconus, a rare disease that impacts the cornea, is no exception. It is best…
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