According to a story from globenewswire.com, the biopharmaceutical company Abeona Therapeutics, Inc. recently issued an announcement in regards to the company's Phase 1/2 clinical trial, which is testing the company's…
According to a publication from ANCA Vasculitis News, a new study in International Kidney Reports suggests that stopping treatment of the disease once it goes into remission may lead to…
The Growth of Precision Medicine Precision medicine is the idea that each patient is an individual, and therefore deserves an individualized treatment plan to secure the best outcomes. It is…
According to a story from Genome Web, a team of researchers from across the country have concluded an in-depth genetic analysis focused on the origins of Hirschsprung's disease, a rare…
May is Myositis Awareness Month! Myositis is the inflammation or swelling of the muscles. There are two primary kinds of myositis: Polymyositis, which causes muscle weakness, usually in the muscles closest…
Thursday May 16th is HAE Day, when we observes the hopes, struggles, and resilience of the Hereditary Angioedema (HAE) patient community! What is HAE? “Angio” means pertaining to blood vessels, and…
According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…
Happy Wednesday! We're officially halfway through May if you can believe it! This week we're highlighting a piece about an intern using their rare experience to inform their work, followed…
According to a story from KUT News, Ava Shepperd and John Ben Shepperd, aged 14 and 18 respectively, always knew that they would eventually need to get kidney transplants. This…
The FDA has just recently approved CYRAMZA as a treatment for those with hepatocellular carcinoma (HCC), who have been treated with sorafenib, and who have ≥400 ng/mL of alpha-fetoprotein (AFP).…
According to a story from mirror.co.uk, Professor Selim Cellek is leading a clinical trial that will test a drug combination designed to treat erectile dysfunction associated with Peyronie's disease, a…
May is Cystinosis Awareness Month! During the month (and all year long!) we strive to bring together the cystinosis patient community and their families -- and the Cystinosis Research Network's…
According to a story from F3News, 77 year old Ron Davis has lived a life highlighted by incredible scientific achievement. He leads the lab that played a critical role in…
According to a story from apnews.com, the drug developer Ayala Pharmaceuticals has recently announced that the company's investigational product candidate, known as as AL101, has earned Orphan Drug Designation. This…
According to a story from Palomar Health, a patient with Hunter syndrome is about to finish up the company's Pathmaker Internship program. The patient in question is Kyle Underwood, who…
Confusion, nausea, fever, anger, then back to normal: this was Lauren Vandiver’s life beginning in early childhood. Her parents, Phillip and Angela Vandiver, spent more than twenty years asking the…
A story from findmecure.com describes several of the latest developments in the area of myasthenia gravis treatment that patients should be aware of. The field of rare disease treatment is…
According to the Wall Street Journal, the National Institutes of Health (NIH) has reversed its initial decision to prevent two of its researchers from meeting with federal investigators to comment…
H.R. 2408 H.R. 2408 is a new piece of federal legislation otherwise known as the "Ensuring Access to Quality Complex Rehabilitation Technology Act." Essentially, this act creates a new category…
According to a story from CityScene, Ohio State University's Dr. Michael Racke is considered one of the country's cutting edge researchers in the field of multiple sclerosis treatment. He is…
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Pharmaceutical companies have spent billions of dollars on Alzheimer’s research. The target has been the toxic protein, amyloid-beta. According to a recent article in NPR News, this theory was…
David Rowland, age 77, is a cancer survivor and former veteran of the Air Force. He is also a tube feeder. People who use a feeding tube are considered disabled…
Scleroderma Scleroderma refers to a whole group of diseases, all rare, that cause the skin and connective tissues in the body to harden. This is a result of excess collagen…
According to a story from Specialty Pharmacy Times, data from a Phase II clinical trial has recently been released which suggests that the drug tagraxofsup is a useful treatment in…
Rare disease patients spend an average of eight years searching for a diagnosis, and for some, that search can last decades. April Hudson of Quinton, Ala., began showing symptoms of…
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