In a news release from late May 2021, biopharmaceutical company Timber Pharmaceuticals, Inc. ("Timber") shared that all patients have been enrolled in the Phase 2b CONTROL trial. During the trial,…
Could a specialized cancer vaccine actually improve patient outcomes? According to a news release from biotechnology company AIVITA Biomedical, Inc. ("AIVITA"), the answer could be "yes." Citing data from a…
Komodo Health and PicnicHealth have just announced that they have partnered together in effort to combine their data and better serve patients. This data will then be made accessible to…
Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger.…
Typically, idiopathic intracranial hypertension (IIH) is associated with obesity, or a body mass index (BMI) over 30.0. As obesity rates have continued to increase globally, so have the incidences of…
Previously, Ocaliva (obeticholic acid) was approved as a prescription treatment for patients with primary biliary cholangitis (PBC), a rare and chronic liver disease. However, Market Screener reports that biopharmaceutical company…
At just four years old, Gabriel Spiro is facing his 3rd open-heart surgery. You see, Gabriel was born with hypoplastic left heart syndrome (HLHS), a rare and severe congenital heart…
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…
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