The Patient Access Network Foundation (PAN) is an organization which works to ensure that patients living with rare, chronic, or life-threatening diseases are not prohibited from receiving treatment due to their…
"Beyond research motivation, I want to give a voice to the disease to help researchers empathize and further understand who all their hard work is going toward." These are words from…
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…
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…
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…
The Kids Wish Network (KWN) works to provide positive experiences for children age 3 to 18 who are living with life-threatening conditions. With the help of various sponsors, they are…
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…
"I feel powerless to help them. There needs to be a change. And change ought to begin in the form of a policy shift." These are Jay Avasarala's words in…
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…
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…
"It helps to share experiences with others who understand what I am going through." Jill Price, a mom with Charcot-Marie-Tooth Disease talks about how social media has helped create a…
Kinetic Kids is a nonprofit which was originally formed to keep children who needed physical therapy active outside of their actual physical therapy sessions by giving them the opportunity to…
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…
Amyotrophic Lateral Sclerosis (ALS) stops communication between the brain and voluntary muscles. Patients slowly lose muscle strength, affecting their ability to walk, talk, and even breathe. The illness eventually results…
People with spinal muscular atrophy and other rare conditions with either few or no approved treatments have recently had their hope of a cure renewed by the concept of gene therapy. Basically,…
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