According to a story in Charcot-Marie-Tooth News, the CMT&Me study has launched a competition in Europe with the purpose of raising awareness and money for CMT and related disorders. Any…
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 many areas of society, from healthcare to big business, companies are turning to artificial intelligence (AI) to solve pressing problems. Within the medical sphere, researchers recently developed an automated,…
From December 1-4, 2020, Reuters hosted a virtual conference called Pharma & Patient USA 2020. The primary focus of this event was furthering patient centricity in disease development as well…
In early December, biotechnology company Aeglea BioTherapeutics, Inc. ("Aeglea") announced that its therapy ACN00177 received Rare Pediatric Disease designation from the FDA for the treatment of homocystinuria. Should this drug…
In a recent press release, biopharmaceutical company Akari Therapeutics ("Akari") announced the publication of data on intravitreal nomacopan for patients with autoimmune uveitis. The data was sourced from a 2-year…
Recently, the FDA expanded treatment indications for Gavreto (pralsetinib). According to MedPage Today, the drug was previously indicated for the treatment of patients with lung cancers with RET gene fusions. Now, this…
In a press release from late November, pharmaceutical company BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from its APeX-J clinical trial in Allergy. During the trial, which was…
A review of COVID-19 data from the UK shows that patients with rare autoimmune rheumatic disease (RAIRD) face a higher mortality risk, according to an article in Healio. The study,…
Xavier Alford told BBC News how he vividly recalls the day, twelve years ago, when he was told by his doctor that he had a very rare disease. His doctor…
A study in the European Medical Journal aims to confirm the safety profile of vedolizumab, a treatment for inflammatory bowel disease (IBD). Specifically, they focused on acute infusion reactions. It…
A webinar hosted by CureDuchenne featuring an informative program by Mesentech is scheduled for 4:30pm EST on Thursday, December 10th. The community is invited to join in and learn directly…
According to a story from Hematology Advisor, a recent study released in Scientific Reports indicates that gene expression has the potential to predict both primary resistance and treatment response in patients living…
Overall, Galapagos works to develop small-molecule treatments for patients with a variety of conditions. In a recent press release, the company reported positive top-line data from the Phase 2 PINTA…
Obesity is considered to be excess fat accumulation that may lead to health issues. Worldwide, the prevalence of obesity has greatly increased over the years; the World Health Organization (WHO)…
According to GuruFocus, the European Medicines Agency (EMA) recently validated a Marketing Authorization Application (MAA) submitted by biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum"). The MAA was centered around maralixibat, an…
On November 30, 2020, biopharmaceutical company Travere Therapeutics ("Travere") announced that patient enrollment was complete for the Phase 3 DUPLEX clinical trial. Within the trial, researchers will evaluate the safety,…
On December 9th, 2020, a special edition of "Behind the Mystery: Rare and Genetic" will air on Lifetime TV as part of the acclaimed morning program The Balancing Act. It…
The FDA has recently granted its Rare Pediatric Disease designation to Moleculin's WP1066 for three indications: atypical teratoid rhabdoid tumor, diffuse intrinsic pontine glioma, and medulloblastoma. This designation will aid…
According to a story from GlobeNewswire, the biotechnology company Galecto, Inc., has recently published the results from its phase 2a clinical trial. This study was testing the company's experimental therapy…
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…
While there is still a lot left to learn about COVID-19, there are a number of facts that we know, one of which is that children face less severe cases.…
According to Newswise, the FDA recently approved a new prostate cancer imaging technique developed by researchers at UCLA and UCSF. The imaging technique, called prostate-specific membrane antigen PET imaging (PSMA…
Axcella is a biotechnology company that has developed a new system of treating complex diseases. Its lead candidates are products that treat two liver diseases, as well as NASH, which…
In a press release from the FDA; the FDA has new recommendations for a common mercury-based filling, dental amalgams. While the mercury in the tooth treatment isn't significant enough to…
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