In a press release from Sanofi, a global biopharmaceutical company, the company's experimental therapy candidate olipudase alfa has demonstrated the ability to substantially improve spleen volume and lung function in…
Colorectal Cancer (CRC) is now the third leading cause of cancer-related deaths in the U.S. A report in Foundation Medicine highlights data on the increase in the number of cases…
According to GlobeNewswire, the results from a Phase 1 study of lirentelimab have been released. Allakos developed this medication as a treatment for eosinophilic and mast-cell related diseases. In this…
According to a story from Medscape, a recent large scale trial focused on cancer treatment has produced some interesting results. The trial was aimed at testing next generation sequencing of…
Researchers from Rensselaer Polytechnic Institute have been conducting research on tau proteins, specifically how it spreads from cell to cell. According to an article in Newswise, this team of researchers…
Currently, there are a number of vaccines for COVID-19 that are being investigated, one of which is AZD1222. The trial was paused back in September when a participant became ill…
A CNBC News item announced a digital chemistry breakthrough that may revolutionize the entire drug industry. The chemists at Glasgow University’s Cronin Lab have created software that sends out instructions…
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…
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…
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…
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…
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 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…
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…
A new study has uncovered that using a hypomethylating agent in combination with a BCL2 inhibitor called Venetoclax may be advantageous as a therapeutic option for individuals with acute myeloid…
Researchers at the Mayo Clinic have collaborated with the international community to develop a potential way to test for spinocerebellar ataxia type 3 (SCA3), also called Machado-Joseph Disease. Additionally, they…
A recent study published in PLOS ONE has reinforced the importance of vaccinations specifically for those already fighting a rare disease. This study was written by Sylvie Lesage from the…
Monitoring tumors is an essential part of cancer treatment; it allows doctors to make informed decisions regarding which therapies to use. For many forms of cancer, medical professionals have been…
A new treatment for Duchenne muscular dystrophy (DMD) has recently been approved by the FDA. The therapy is called viltolarsen and it was created by Nippon-Shinyaku and the National Center…
A recent study has found a 12.5% improvement for patients with severe COVID-19 when baricitinib was used in combination with remdesivir. That means a faster recovery time for patients and…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
