Microcephaly is caused by a depletion in neural progenitor cells (NPCs). This depletion is caused by a mutation in the centrosome genes, and it occurs while the brain is still…
In a recent press release, biopharmaceutical company Junshi Biosciences ("Junshi") announced a burgeoning collaboration with leading biologics platform company Coherus BioSciences, Inc. ("Coherus"). The pair is working to develop and…
Last year, 92.7 WOBM reporter Vin Ebenau sat down with Allie and Jonna Crocker, two sisters who live around the Jersey Shore, to discuss their experience with rare diseases. The…
As reported in Charcot Marie Tooth News; researchers have devised a new measure of quality of life for youth with Charcot-Marie-Tooth disease. While of course, researchers could assess the youth’s…
In a recent press release, biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum") shared that the company finished its rolling New Drug Application (NDA) submission. The NDA focuses around maralixibat, an investigational…
GeneDx, an OPKO Health company, has recently launched several genetic tests. Within this group of tests are repeat expansion analysis for Friedreich ataxia, spinocerebellar ataxia (SCA), and other forms of…
Okay, I'll admit it -- I'm a bit of a reality TV junkie. So I have been watching Cheyenne Floyd's journey since her initial entrance on Are You The One? Season 3…
Charlie Fry is a three-year-old from Kansas City living with SLC6A1. His family is very active in the SLC6A1 community, even starting a nonprofit titled "A Cure for Charlie." They…
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…
Recently, biopharmaceutical company Soligenix, Inc. shared that it had been granted a patent (#16102842.8) in Hong Kong for the use of synthetic hypericin to treat patients with cutaneous T-cell lymphoma…
Poly (ADP-Ribose) polymerase inhibitors (PARP inhibitors) are used to treat neoplasms. They are frequently used as a therapy for ovarian cancer. A recent study has attempted to detect the possibility…
The China Center for Drug Evaluation (CDE) has recently granted Harbour BioMed the Breakthrough Therapy designation for batoclimab, a treatment for adults with myasthenia gravis (MG). This designation was created…
Tara Johnston, a resident of London, England is now twenty years old but still coping with severe acne. Tara interviewed with MyLondon in an effort to raise awareness and share…
Monogram Health has recently announced that they will be expanding their collaboration with Humana Inc. to provide greater access to chronic kidney disease and end-stage renal disease treatment. They will…
Unfortunately, a post-marketing safety study did not go so well for Pfizer, whose drug XELJANZ (Tofacitinib) failed to meet one of its co-primary endpoints. Biospace explains that XELJANZ is designed…
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 press release from late January 2021, biopharmaceutical company Seelos Therapeutics, Inc. ("Seelos") announced the issuance of an Australian patent for Trehalose (SLS-005). The patent (#2019204513) centers around the…
According to a recent press release, pharmaceutical company Karyopharm Therapeutics Inc. ("Karyopharm") achieved a milestone in the NEXPOVIO (selinexor) drug development process. The Committee for Medicinal Products for Human Use…
According to a press release from Sanofi, platform and poster presentations focused on the company's investigational product candidate avalglucosidase alfa are slated to be made public at the 17th annual…
In a news release from biopharmaceutical company Bristol Myers Squibb, the company shared that the FDA accepted its supplemental New Drug Application (sNDA). Because the sNDA was submitted alongside…
At the start of February, gene therapy company Spark Therapeutics ("Spark") announced that the first participant in the RESOLUTE trial was dosed with SPK-3006. The trial is evaluating the…
A study has recently highlighted the fact that patients who have type 2 diabetes are more susceptible to chronic kidney disease (CKD), and as a result, cardiac issues. This study…
The month of February is recognized annually as Turner Syndrome Awareness Month, which is intended to help spread awareness about Turner syndrome among the general public and in the medical…
Sometimes it can be difficult to study or learn about rare diseases, especially when there are not many patients. For example, only an estimated 30,000 Americans have Castleman disease. Yet…
The very first patient has just been dosed in Pfizer's Phase 3 clinical trial for Duchenne muscular dystrophy (DMD). The trial is called CIFFREO. This patient received the therapy at a…
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