Chronic kidney disease (CKD) affects approximately 26 million people in the United States. CKD usually develops after a patient has already battled a different illness which has placed a toll on…
Apellis Pharmaceuticals has just announced that they have received Orphan Drug Designation from the FDA for APL-2, a C3 complement inhibitor. The company is currently investigating the drug as a…
Nonalcoholic steatohepatitis (NASH), is condition most commonly caused by obesity or type 2 diabetes. It can result in fibrosis, complications in the lung and cardiac system, liver cancer, liver cirrhosis,…
Paroxysmal nocturnal hemoglobinuria (PNH) prematurely destroys the bodies red blood cells (hemolysis). It can cause severe anemia, kidney disease, fatigue, as well as other symptoms. Up until now, there has…
According to a story from ksl.com, a recent poll found that the majority of Americans are in support of using gene editing technology in order to protect newborns from genetic…
Happy New Year! For our first Editor's Choice of 2019, we want to highlight four articles from the last couple weeks. First, we have the story of young man who…
Urea Cycle Disorders (UCD) are rare genetic conditions caused by an enzyme deficiency. The urea cycle is responsible for removing excess levels of ammonia from the body. Individuals with UCD…
Lichen sclerosus is a rare, under-researched, and poorly understood condition. The lack of knowledge concerning the disease has led to a lack of awareness, late diagnosis, and a limited amount of…
A Fanconi Anemia (FA) investigative gene therapy now has PRIority MEdicines (PRIME) designation from the EMA. This designation helps to accelerate the development of therapies for patient populations who have…
Cold agglutinin disease (CAD) occurs when the body's antibodies destroy their own red blood cells. It is a form of hemolytic anemia, meaning the body can't produce enough blood cells fast…
Dermatomyositis (DM) is a disease which causes muscle weakness and rashes on the skin. Immunosuppressive drugs are a common treatment prescribed for the condition. Patients may also be given methotrexate, rituximab,…
The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Patient Worthy. According to a story from Vox, a review of over…
Pantothenate kinase-associated neurodegeneration (PKAN) is a neurodegenerative disorder caused by a mutated PANK2 gene. This mutation disrupts a metabolic pathway, decreasing the body's levels of CoA. CoA, or coenzyme A,…
According to a story from Scoop Independent News, citizens of New Zealand that have the rare bleeding disorder hemophilia A will soon have access to the drug emicizumab (marketed as…
According to a story from globenewswire.com, the gene therapy company AVROBIO recently announced that the company's experimental gene therapy drug candidate AVR-RD-01 has earned Orphan Drug designation from the US…
According to a story from BioSpace, the biopharmaceutical company Merck recently announced that the US Food and Drug Administration (FDA) has approved the company's cancer drug KEYTRUDA in a new…
Smith-Magenis Syndrome (SMS) is caused by a deletion of the 17p 2,3 chromosome. the condition affects 1 in every 15,000-25,000 individuals. It often results in a sleep disorder, and various mental, physical,…
Leukodystrophy is the term used for a group of rare, progressive brain disorders which cause the brain's myelin sheath (the nerves protective covering) to either develop improperly or be destroyed…
Methylmalonic Acidemia (MMA) is a liver condition which affects the body's ability to break down food and fatty acids. A dietary imbalance or minor virus for these patients may spark a metabolic…
Nontuberculous mycobacterial (NTM) lung infection occurs when someone inhales nontuberculous mycobacteria from the environment. There are various types of this bacteria, giving NTM different forms. But in all types of NTM,…
Morgan's story Morgan Gleason is a 20-year-old living with Juvenile Dermatomyositis. It's a rare autoimmune condition causing weak muscles and rashes in addition to other symptoms. As a result of her…
According to a story from thenewsreports.com, the biopharmaceutical company XORTX Therapeutics, Inc., recently announced that the company and its partner Cato Clinical Research have received correspondence from the US Food…
A diagnosis means everything for a rare disease patient. However, to process 200 patient records manually, it would take the most dedicated physician approximately 40 hours. That's one full work…
According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…
Immune Thrombocytopenia (ITP) is diagnosed by low platelet counts in the blood. It also causes impaired platelet production. This put patients at risk for serious bleeding as well as spontaneous bruising.…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
