The Bausch + Lomb Corporation has just released new findings from a survey of retina specialists. Their goal was to understand the need for some type of new treatment for…
According to a story from Medpage Today, a recent study found that a dual immunotherapy approach could be an effective treatment for cervical cancer that had been previously treated, has…
On May 25, 2022, the Rare Disease Legislative Advocates (RDLA) held a briefing for the Rare Disease Congressional Caucus. This virtual event provided an update on various legislative initiatives relevant…
In Detroit, activists recently came together to raise money for pancreatic cancer research through a walk called “The PanCAN PurpleStride walk.” Each year about 62,000 people are newly diagnosed with pancreatic…
Tourette Syndrome affects one million Americans. 2022 is the Tourette Association of America's 50th Anniversary, and to celebrate, you can get involved in Tourette Syndrome Awareness Month from May 15th…
HAE Junior, an advocacy group from the Czech Republic and a Patient Worthy partner organization, is issuing a renewed call for submissions for its Stronger Than HAE art exhibition. This…
In Europe, Orphan Drug designation is granted to therapies intended to treat, prevent, or diagnose a rare, life-threatening, or debilitating disease. For the purpose of this status, rare conditions…
According to a story from the Children's Tumor Foundation (CTF), the month of May is recognized as Neurofibromatosis (NF) Awareness Month. This is a time for spreading awareness about neurofibromatosis…
According to a story from Globe Newswire, The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently announced a positive opinion in regards to the experimental…
Ala Moshiri from the University of California is both a researcher and an eye doctor. He recently discussed his frustration surrounding the lack of available treatments for so many genetic…
The New Era for Preventing End-Stage Kidney Disease Act (H.R. 7506) is a new Act sponsored by Reps Gus Bilirakis and G.K. Butterfield. This Act would transform care for those…
According to a story from ALS News Today, around 5,000 people get diagnosed with amyotrophic lateral sclerosis (ALS) annually. In addition, costs of treatment for patients average $250,000 out of…
The month of May is recognized as National Myositis Awareness Month, a time to spread awareness about myositis among the general public and the medical field. Organized by The Myositis…
Al Lupiano, an environmental scientist, recently spoke with Meg Baker of CBS2. Al told her that twenty years ago, he was diagnosed with a brain tumor. The memory and…
Living with a rare disease is difficult for so many reasons. One of the lesser talked about struggles of living with a rare condition is the fact that so few…
PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease where blood cells break apart and cause the contents to circulate freely within the bloodstream. This condition is caused by a…
International Fibrodysplasia Ossificans Progressiva (FOP) Awareness Day is recognized each year on April 23rd. This is a time for spreading awareness about this rare disorder among both the general public…
According to a recent article, researchers at the WORLDSymposium revealed they are looking into using pentosan polysulfate sodium to treat Maroteaux-Lamy syndrome. MPS VI (Maroteaux-Lamy Syndrome) Maroteaux-Lamy syndrome is…
A team of researchers from Yale School of Public Health, including Dr. Peter Krause, Dr. Durland Fish, Demerise Johnston, and others, have published a study in the journal Clinical Infectious Diseases documenting a…
According to a story from Rolling Stone, iconic comedian Gilbert Gottfried has passed away from complications related to myotonic dystrophy type II, a rare disease. His publicist disclosed his cause…
In the past, filgotinib (Jyseleca) has largely been used to treat patients with rheumatoid arthritis (RA). However, researchers hypothesized that the treatment could be beneficial in other indications. Following…
According to an article recently published in Business Wire, Global Genes, a leading advocacy group, released a progress report on rare diseases. The report includes data and studies as…
Note: this story was originally published by HAE Junior, a Patient Worthy partner Interview with Malena Vetterli from the European Reference Network (ERN-RITA) for rare immunodeficiencies, autoinflammatory and autoimmune diseases.…
According to a story in the WSJ, the FDA has approved a new therapy for multiple myeloma patients. The therapy is called Carvykti and it is now available as a…
According to a story from Targeted Oncology, the company Genmab has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to its investigational therapy…
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