Tanner McLeod has been searching for a stem cell donor for a long time. He was diagnosed with sideroblastic anemia at just six months old and has required a transplant…
Recent results of a Turkish trial have demonstrated the need for further research into IFN-gamma as a treatment for Friedreich's ataxia (FA). This study showed that the therapy resulted in…
While gene therapy is a burgeoning technique to treat a number of rare genetic conditions, there are still a few complexities associated with this treatment option. How safe and effective…
Over the next 2-2.5 years, biotech spin-out company Augustine Therapeutics will be working to understand both the underlying biology of Charcot-Marie-Tooth disease (CMT) and potential therapeutic options for patients.…
Rare Disease Day each year on February 28th provides a time for the rare disease community to reflect on the progress of the year, the continued gaps in rare disease…
MSN News recently interviewed Dr. Laxmikant Palo, with the conversation focusing on the situations of people living with rare diseases in India and how to improve them. According to Dr.…
For over 15 years, researchers and scientists have been working to understand the underlying genetics of macular telangiectasia (MacTel), a rare and debilitating eye disease, through the MacTel Project.…
For years, scientists have known the mechanisms underlying normal, as well as the disease-causing (pathogenic) versions, of prions. But according to a recent article published in Sci-Tech Daily, no one…
As reported in PR NewsWire; this March, Calliditas Therapeutics AB announced they have submitted a New Drug Application (NDA) for their novel oral solution, Nefecon, to treat primary IgA Nephropathy…
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 study from hindawi.com, the serum glycocholic acid (GCA) to total bile acid (TBA) ratio can be used to identify people living with nonalcoholic fatty liver disease (NAFLD).…
George Mendoza has inspired others throughout his whole life. He has been an athlete, an author, a motivational speaker, and more. Born in 1955 in New York City, Mendoza has…
Despite the pandemic, there were many strides in the field of medicine and research in 2020. For example, researchers first discovered and identified a rare disease called VEXAS syndrome. The…
Marian McGlockin was eighteen months old when she was diagnosed with Niemann-Pick Type C disease, a rare and fatal disorder. Marian could barely speak and was unable to walk. According…
Charcot-Marie Tooth disease (CMT) may be the most common inherited disorder of the peripheral nervous system, but that only puts the official patient count in Denmark at about 22.5 cases…
Recently, the Endocrine Society's virtual Annual Meeting, dubbed ENDO 2021, took place from March 20 - 23, 2021. During the meeting, attendees discussed the latest in endocrinology research, treatment,…
At the Endocrine Society's Annual Meeting, or ENDO 2021, Amolyt Pharma ("Amolyt") shared positive clinical data from a Phase 1 clinical trial evaluating AZP-3601 for patients with hypoparathyroidism. Although…
According to a story from Medical Xpress, an uncommon fungal infection has been discovered in Alberta, Canada. This infection, called histoplasmosis, is transmitted through the fecal matter of bats and…
In a press release from early March 2021, cell therapy company CiRC Biosciences, Inc. ("CiRC") shared that its chemically induced photoreceptor-like cells (CiPCs) received Orphan Drug designation from the FDA.…
A recent study published in Arthritis Care & Research illustrated how higher dosages of prednisone lead to better outcomes for lupus nephritis (LN) patients. After one year, participants were found to…
In a study published on pubmed.gov, an investigation of 100 patients living with progressive supranuclear palsy, a rare disorder, has helped to confirm the usefulness of a diagnostic approach called…
No longer the new player on the team, next-generation sequencing (NGS) is well established as having an influence in clinical care. In recent years, NGS has been responsible for many…
Back in October, the International Pemphigus & Pemphigoid Foundation (IPPF) took another step in their plan to learn from and work with patients to increase awareness for pemphigus. They held…
A Phase 3 clinical trial run by Pharnext for Charcot-Marie-Tooth disease Type 1A (CMT1A) has just been awarded 13.3 million dollars. $7.2 million comes from existing shareholders and $6 million comes…
Rare disease therapies have an exorbitant cost. Fewer patients means higher costs in order to support the development of treatments. In the United States, 966 billion was spent in 2019…
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