WKBW Buffalo recently interviewed ALS patients who are advocating for a bill that would grant access to clinical trial drugs during Phase III and before they go through the…
ANCA-associated vasculitis (AAV) generally targets two enzymes, myeloperoxidase (MPO) or proteinase 3 (PR3) located in the granules of monocytes and neutrophils. These enzymes play a critical role in the…
Kyleigh Kista, a 3 year old from Benonin South Africa needs heart surgery within the next 3 months to save her life. Kyleigh was diagnosed with tetralogy of Fallot with…
The Myrovlytis Trust, a UK Charity focused on promoting research and advancing education for rare diseases, and Patient Worthy are now partners! This organization has focused primarily on Birt-Hogg-Dubé syndrome…
Non-clear cell renal cell carcinoma (nccRCC) has recently been found to have a very strong connection with various subtypes of proliferative disease. Further, this condition was found to have a…
The FDA grants the Orphan Drug designation to treatments indicated for rare diseases, which are defined as affecting less than 200,000 people in the U.S. This designation is meant to…
For many years, scientists have known about toxic clumps forming in patients with Huntington’s disease (HD). However, until recently, scientists relied on examining post mortem brain samples of HD…
Dr. Richard Stone recently interviewed with Targeted Oncology to discuss challenging acute myeloid leukemia subtypes and a list of available therapies. Dr. Stone was asked to look back at…
Discovering a treatment for a rare disease is challenging and rewarding, especially when it is an extremely rare disease such as perivascular epithelioid cell tumor (PEComa). Biospace recently carried…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
For those of us who suffer from depression, that mostly invisible mental illness that, at its worst, can render us incapable of getting out of bed and, at its best,…
Dr. Deborah M. Stephens from the Huntsman Cancer Institute recently presented at the 39th Annual Chemotherapy Foundation Symposium Innovative Cancer Therapy for Tomorrow (CFS). The presentation surrounded how critical identifying…
In June 2021, Health Canada approved TRIKAFTA (elexacaftor/tezacaftor/ivacaftor) for patients with cystic fibrosis (CF), with at least one F508del mutation, aged 12+. According to a news release from late…
According to a recent article, a study shows the risk of getting nasopharyngeal carcinoma (NPC) varies considerably in different ethnic groups among Asian Americans. Nasopharyngeal Carcinoma (NPC) Nasopharyngeal carcinoma…
Even as COVID-19 cases die down, post-COVID syndrome still plagues many individuals across the world. These individuals have faced ongoing COVID-19 symptoms for more than two weeks following diagnosis. The…
On November 18, 2021, the Rare Disease Legislative Advocates (RDLA) held their monthly webinar. These webinars provide the rare disease community with updates regarding relevant legislation. As the year begins…
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…
Claire Richmond lives with acute hepatic porphyria (AHP). She searched for 19 years to find answers to her unexplained pain. She wasn't diagnosed until she was 32. She recently had…
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…
CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…
According to a recent article, PharmaTher Holdings Ltd. announces the FDA granted orphan drug designation (ODD) for ketamine in the treatment of complex regional pain syndrome (CRPS). Complex Regional…
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…
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 Rare Disease Diversity Coalition and Global Genes have recently established a new partnership. This partnership aims to help find solutions to help address outcome disparities faced by people of…
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