Earlier this week, biotechnology company Syntrix Pharmaceuticals ("Syntrix") announced that it had finished dosing all patients in its Phase 1/2 clinical trials. The trials were designed to evaluate SX-682 as a…
A recent study, published in the journal Medicine, has found that Mycopheenolate mofetil (MMF) could be a more advantageous therapy for lupus nephritis than cyclophosphamide (CYC) when it comes to improving serum complement…
While Lyme disease is one of the better known tick-borne illnesses, many people might not know about Powassan virus. This flavivirus, common in both Russia and North America, is found…
Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) The RDCA-DAP, funded by the FDA, was first launched in September of 2019. Its aim was to make data more accessible for…
Not all treatments work for every patient, with rheumatoid arthritis (RA) medications being the perfect example. Disease-modifying anti-rheumatic drugs (DMARDs) are the standard of care for RA despite the fact…
Researchers from Nanoscope, thanks to funding by the NIH, have developed a protein that can effectively restore vision in mice. This study was published in Nature Gene Therapy. The Study…
The mission of biopharmaceutical company TG Therapeutics, Inc. ("TG") has always been to create treatments to fulfill unmet needs for patients. Their focus is on autoimmune disorders and B-cell malignancies.…
In late October, commercial-stage biopharmaceutical company Omeros Corporation ("Omeros") announced the publication of data from its narsoplimab clinical trial. During the trial, researchers evaluated the therapy as a treatment for…
Recently, IM Therapeutics, a developer of therapies for patients with autoimmune conditions, announced the start of a Phase 1b clinical trial designed to evaluate IMT-002 for type 1 diabetes. Interestingly,…
According to new research, children with juvenile idiopathic arthritis (JIA) are more likely to experience uveitis flares as their arthritis progresses. Medscape notes that researchers wanted to understand how and…
Many rare diseases are in dire need of need of viable treatments. To fix this problem, researchers have began creating and indicating existing treatments for rare diseases. An example would…
According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan…
As reported in Cushing's Disease News; a woman diagnosed with Cushing's Disease via hair cortisol analysis is being used as a case study for why hair is a good tool…
An article recently published in Genetic Engineering & Biotechnology News, heralds DrugCell, an experimental artificial intelligence (AI) system. DrugCell was created at the Moores Cancer Center and the School of…
In late October 2020, Homology Medicines ("Homology") announced its development of a new gene therapy program to treat patients with MPS II, or Hunter syndrome. Homology's mission is to meet…
According to a story from Biotech 365, the biotechology company Kezar Life Sciences, Inc. recently announced that its experimental therapy KZR-616 has earned Orphan Drug designations from the US Food…
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…
A new treatment for Krabbe disease, PBKR03, has recently been granted the Orphan Drug and Rare Pediatric Disease designations by the FDA, according to an article in GlobeNewswire. These designations…
According to a story from Myasthenia Gravis News, a recent report describes the first-ever documented case of a patient developing the ocular variant of myasthenia gravis in connection with COVID-19…
Happy Friday! November 1 is International Acromegaly Day. For those unaware, Acromegaly is a rare condition characterized by the presence of an elevated level of growth hormone and typically effects…
According to CureDuchenne, two companies have began a collaboration in an effort to create new gene therapies for Duchenne muscular dystrophy (DMD). Ultragenyx and Solid Bioscience are both using their…
Last week, from October 22nd through the 25th, marked the American Society of Nephrology (ASN) Kidney Week. According to a press release, Synthetic Biotic medicine developer Synlogic, Inc. ("Synlogic") presented…
2326 words (source) vs. 503 words (mine) - 6% match Near the start of October, global biopharmaceutical company Jazz Pharmaceuticals ("Jazz") announced positive safety and efficacy data from their Phase…
Recently, specialty pharmaceutical company Xeris Pharmaceuticals ("Xeris") announced that its injectable treatment XP-0863 received Fast Track designation for the treatment of patients with acute repetitive seizures. Xeris created this treatment…
Early on October 28, 2020, Insmed Inc. ("Insmed") announced that it received Marketing Authorization from the European Commission for ARIKAYCE. This therapy is designed for adult patients with nontuberculous mycobacterial…
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