Tyrosinemia is a metabolic disorder which prevents the body from processing the amino acid tyrosine. Without treatment, the disorder can be lethal. There are three different types of tyrosinemia with…
Chronic kidney disease patients often develop aortic stenosis, which requires a transcatheter aortic valve replacement (TAVR) procedure to treat. The question arises: will a TAVR procedure worsen the renal function…
According to a recent article in MedicalXpress, in 2004 Professor Sophie Lucas of the Louvain de Duve Institute began studying immunosuppressive cells that are known to block immune responses in…
According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…
Dane Tidwell lost his leg to osteomyelitis when he was just 18 years old. He spoke about the confusion and loneliness that came with being an amputee, and how his…
According to a story from BioSpace, the biopharmaceutical company Aridis Pharmaceuticals, Inc., has recently announced that it has struck an agreement with the US Food and Drug Administration (FDA) in…
Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001,…
It didn't take long for Jeff and Katis D'Angelo to realize that something was up with JJ, their newborn son. The biggest indicator that something was off was that the…
Josephine Bila was born with transfusion dependent beta thalassemia, a rare blood disorder. Recently, Josephine uploaded a video to Youtube in which she talks about her life as a patient,…
An article that appeared recently in Psychology Today asks whether dementia is responsible for falls. The answer brings to mind the many medical and environmental factors that affect the elderly…
September is recognized as Charcot-Marie-Tooth disease (CMT) Awareness Month. Although this illness is one of the most widespread inherited genetic disorders, there is still a significant need to spread awareness…
According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…
According to a story from BioSpace, Ziopharma Oncology, Inc. has recently announced that its investigational therapy Ad-RTS-hIL-12 with veledimex (also called Controlled IL-12) has been given Rare Pediatric Disease designation…
Mononine, a treatment for hemophilia B, hit the market nearly three decades ago in 1992. Since then, there have been amazing advancements in the knowledge and treatment for this disease.…
According to a story from BioSpace, the biopharmaceutical company Clene Nanomedicine, Inc., has recently announced that it has completed the process of enrollment patient participants in its upcoming phase 2…
According to a story from BioSpace, the biopharmaceutical company Zogenix, Inc., has recently announced the release of results from a phase 3 clinical trial testing the company's drug fenfluramine (marketed…
As reported in BioSpace; today, Biocryst Pharmaceuticals has been given a boost to further their work to explore the merits of a potential oral treatment for PNH. The U.S. FDA…
According to a story from BioSpace, Takeda Pharmaceutical Company Ltd. recently announced the release of results from a phase 3 clinical trial. This trial was testing the company's drug ixazomib…
This year, the Digital International Liver Congress took place online from August 27-29, thanks to COVID-19. During the conference, biopharmaceutical company Mirum Pharmaceuticals announced data from its Phase 2 INDIGO…
According to a story from Newswise, September is recognized as Pulmonary Fibrosis Awareness Month. This includes idiopathic pulmonary fibrosis (IPF), or disease without an identifiable cause. In the midst of…
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…
According to a story from BioSpace, Genentech (of the Roche Group) has announced its plans to present new data related to its drug satralizumab-mwge (marketed as Enspryng™) at the upcoming…
As reported in Biospace; You probably know that a purple ribbon is used to commemorate cancer, pink for breast cancer, orange for leukemia. What do you know about childhood cancers…
According to Charcot-Marie-Tooth News, patients with Charcot-Marie-Tooth disease type 1A (CMT1A) have an extra PMP22 gene. However, new research shows that this excess gene causes a toxic accumulation of protein within cells.…
Although we are now months into the global pandemic, new information emerges daily. Currently, COVID-19 is responsible for over 29 million cases globally, with 926,000 associated deaths. In the United…
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