According to a story from finanznachrichten.de, the biotechnology company Saniona recently announced that its treatment period for adolescent patients in its Phase 2a clinical trial testing Tesomet as a treatment for…
According to a story from drugs.com, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that is had recently completed a end of Phase 2 meeting with officials from the US…
A new clinical trial which will test a two-part combination treatment for Erdheim-Chester disease was recently added to the clinicaltrials.gov website. This trial will be specifically for patients with BRAF…
Prader-Willi Syndrome (PWS) is caused by deleted genes on chromosome 15. This results in improper signaling of satiety, leading to obesity. Individuals with the condition can suffer from respiratory/cardiac disease, gastric…
According to a story from Medical Xpress, a team of researchers affiliated with the Johns Hopkins Kimmel Cancer Center found some encouraging results when they tested an immune system enhancing…
Myasthenia Gravis (MG) is a progressive autoimmune disorder which results in the weakening of muscles. Most people with the condition are able to manage their symptoms with treatment and have…
According to a story from drugs.com, the biopharmaceutical company Catabasis pharmaceuticals recently provided updates on the status of its Phase 3 clinical trial testing its investigational therapy CAT-1004, also known…
A new bronchiectasis research study was added to the clinicaltrials.gov website on New Years Day. This observational study, called the BRIDGE Study (Bronchiectasis Research Involving Databases, Genomics, and Endotyping) is…
When you're told your child has a rare, life-threatening condition you exhaust every resource trying to find answers. But the rarer the condition, the harder those answers are to find…
According to a story from EurekAlert!, the results of a recent Phase 3 clinical trial suggest that the targeted therapy sorafenib could be an effective treatment option for desmoid tumors.…
Batten Disease There are 13 different forms of Batten Disease defined by the medical community. Each presents similarly but there are differences in the severity of symptoms as well as the…
According to a story from Gizmodo, the genetic mutation which causes certain types of dog breeds, such as Boston terriers and bulldogs, to have spiral shaped, corkscrew tails appears to…
Neuromyelitis optica (NMO), or Devic's disease is a rare autoimmune condition which causes inflammation in the optic nerve and/or spinal cord. The condition affects 5 in every 1,000,000 people and…
Limb Girdle Muscular Dystrophy Type 2D (LGMD2D), also called alpha-sarcoglycanopathy, is a form of muscular dystrophy which is caused by a defected SGCA gene. It causes the body to lose function of…
CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It's called CTX001, a type of stem cell therapy. This drug has…
Thanks to a Phase 2 trial, pediatric patients with Philadelphia chromosome-positive acute lymphoblastic leukemia or Ph+ ALL, now have a new treatment option. The FDA has announced approval of Sprycel…
It has just been announced that Capricor Therapeutics has put their Duchenne Muscular Dystrophy (DMD) clinical trial on hold. Fortunately, the company does plan on continuing the trial. What is…
PPIs or Proton Pump Inhibitors are one of the top 10 selling generic drugs in the United States. They're used to treat heartburn and acid reflux by suppressing acid production…
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…
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…
According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently announced that the first patient has been treated in the company's Phase 3 clinical trial testing sparsentan as…
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…
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