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…
New Trial A Phase 1A trial for GMA301 was completed this year in Australia for pulmonary arterial hypertension (PAH). This trial demonstrated a positive safety profile for a range of…
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…
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,…
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…
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…
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 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…
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…
Colton’s Story Debbie Moore was 18 months old when she was diagnosed with X-linked hypophosphatemia (XLH) a rare, deforming, and painful bone disorder. XLH causes softening of the bones…
A recent article in Medical Express reminded readers that our DNA goes back many generations, yet it was possible for a genome archeologist to call upon ancient elements that are…
Novartis has recently released interim data on iptacopan, according to a press release. The data on the C3 glomerulopathy treatment was released at the American Society of Nephrology 2020 Annual…
Dr. David Pisetsky, a translational researcher at the Medical Center of Duke University, recently described his team’s new approach to treating potential Lupus patients. The doctor told the Rheumatologist of…
The documentary "Walk in My Shoes” takes viewers through a lifetime with cystinosis with Chandler and his family. After Chandler was diagnosed with the rare disease as an infant, making…
According to a story from SMA News Today, the Patient Access Network (PAN) Foundation announced that it is now offering financial assistance to help patients living with the rare disease…
The FDA has officially approved remdesivir for the treatment of adults and children (over the age of 12 and weighing at least 40 kg) who have been hospitalized for COVID-19.…
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