Jeffrey Miner, Ph.D., Director of Basic Research in Washington University’s Division of Nephrology, has received a $2.25M five-year grant from the National Institute of Diabetes and Digestive and Kidney…
Mereo BioPharma Group has just announced that they have received Orphan Drug Designation from the FDA for their new treatment for alpha-1 antitrypsin deficiency (A1AD) called alvelestat. This therapy is…
Results from a study on systemic mastocytosis (SM) have recently been published. This investigation aimed to understand the prevalence of contrast media induced hypersensitivity reactions (CMIHR) in different subtypes of…
When you are looking for new treatments that will either stop or slow the development of Alzheimer’s disease, where would you look? According to a recent article in Gladstone…
The FDA, the NIH, 5 nonprofits, and 10 pharmaceutical companies, have formed an official partnership to accelerate gene therapy development for rare diseases. Out of 7,000 known rare diseases, just…
The Vestibular Disorder Association (VeDA) has just published a guide to handling the holidays when you are a vestibular patient. Holidays can be a challenge because of overstimulation, fatigue, high…
Dr. Farah Naz Khan’s recent article for GoodRx Health attempts to explain the difference between Hashimoto’s disease and hypothyroidism. The terms are used interchangeably but Dr. Khan’s article explains…
According to a recent article from BioSpace, Boston’s Children Hospital was granted $308,000 to advance their multidisciplinary biomarker work. SYNGAP1 Syndrome SYNGAP1 syndrome is a neurological disorder that is characterized…
The Times Now News recently featured an article that highlighted a study by a group of NYU Langone scientists. The study paper was published October 6th, 2021 in the…
Albert Edge and Yushi Hayashi recently led a research team which uncovered the specific mechanism which can cause deafness in Norrie disease, a rare condition. These findings were recently published…
The University of Pittsburgh School of Medicine along with Prairie View A&M University, have recently uncovered that there are some cancer drugs which could be used as an effective treatment…
Aurion Biotech has just released the results from their clinical trial called IOTA. This trial was conducted by researchers in Japan and demonstrated that corneal endothelial cell therapy was able…
Healx has just announced that they've been given Investigational New Drug FDA approval for HLX-0201 for Fragile X syndrome. This means they are able to move forward with their Phase 2a…
The COVID-19 pandemic has led to many changes, disruptions, and setbacks. For rare diseases, it did delay research for new treatments. However, there were a few positive things that came…
A new article has been published in Neurology and Therapy which specifically discusses the lived experiences of patients with myasthenia gravis (MG). The most notable thing about this article is that…
Three doctors recently got together to discuss how they manage care for their sickle cell disease (SCD) patients. As a part of this conversation, they discussed how they combat systemic…
Rare Diseases South Africa (RDSA) and The Rare Diseases Access Initiative (RDAI) have recently come together to talk about the issues that those living with rare diseases in South Africa…
Maya Doyle is a health social worker specifically focused in pediatrics and the transition from pediatric care to adult care. She has been practicing for twenty years. Now, she works…
In a news release from September 30, 2021, biopharmaceutical company Arrowhead Pharmaceuticals ("Arrowhead") shared that it had initiated its Phase 2b AROAPOC3-2002 clinical trial and that the first patient…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Japan's Ministry of Health, Labour, and Welfare has just announced its approval of Alofisel, a stem cell therapy created by Takeda. This approval is specifically for patients who are diagnosed…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
CurePSP has just announced their latest Venture Grant recipients. These grants total $320,000 and will all support the research of neurodegenerative disease treatments. CurePSP gives these grants twice every year.…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Science Direct recently published a Respiratory Medicine Case Report that may impact future Cystic Fibrosis (CF) treatment. The case involves adult brothers herein referred to for purposes of this…
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