According to a story from GlobeNewswire, the medication dupilumab (marketed as Dupixent®) has demonstrated potential efficacy in a phase 3 clinical trial as a treatment for eosinophilic esophagitis, a rare…
A recent article published in Multiple Sclerosis News Today reported that newly identified immune cells prevented the death of injured nerve cells. A study finds that this particular immune cell…
A rare disease diagnosis is often shocking, and it means making life changes. An adrenoleukodystrophy (ALD) diagnosis is no exception. Because this condition typically impacts children, parents have to make…
Medical professionals have been studying inflammatory bowel disease (IBD) for many years in an effort to fully understand it. Now there has been a breakthrough, as researchers from the Children's…
Across the world, various countries have Orphan Drug or Orphan Medicinal Product designations that leading agencies grant to products designed to treat patients with rare diseases. Recently, AVROBIO announced that…
Positive results have just been announced for a Phase 2 study called QUARTZ2, investigating SHR0302 as a therapy for atopic dermatitis (AD). Researchers found that at every dose level examined,…
Medical professionals are constantly looking for treatments that will halt or even reverse the progression of Alzheimer's disease, and a new vaccine called E22W42 may be a large step in…
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…
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…
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…
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 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…
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…
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…
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…
In a recent study published in Clinical and Experimental Immunology, researchers determined that patients with high ANCA antibody levels are at an increased risk of having ANCA-associated vasculitis (AAV) relapses. According…
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