A phase 3 clinical trial has produced positive results for cutaneous T-cell lymphoma (CTCL) patients. The drug tested is called brentuximab vedotin (Adcetris). Specifically, the drug was created to treat…
According to CBC, Marie-Eve Chainey was on dialysis six out of seven days a week. For 15 years, this was her life. It was a necessary feat to control her…
Case Western Reserve University researchers have recently developed gene-carrying nanoparticles that are able to bring healthy genes to those negatively affected by Leber congenital amaurosis (LCA), a disease that causes…
The Pulmonary Fibrosis Warriors (PF Warriors) and monARC Bionetworks teamed up today to host a Facebook Live video, sharing important information about how patients can become more involved in research…
The Associated Professional Sleep Societies held their 31st annual meeting in Boston June 3rd through 7th. You can read more about Sleep 2017 by clicking here. Among other discussions was cataplexy.…
In April, Prometic Life Sciences Inc. announced it would be submitting a Biologics License Application to the FDA for treatment of hypoplasminogenemia. The company's therapy has already been listed with Orphan…
The goal of the Alpha-1 Foundation is ultimately to find a cure for Alpha-1 Antitrypsin Deficiency (A1AD). But along the way, they are also a resource for patients, families, and…
Gastroparesis causes paralysis of the stomach. It basically means the stomach doesn’t understand its job, and can’t digest food properly. This causes extreme discomfort (to say the least) for gastroparesis…
Andrew Belliveau started the Pie Face Challenge in July of 2016 in order to raise awareness for gastroparesis and stimulate accelerated research for the disease. He took inspiration from the…
Achondroplasia is one of those illnesses that you can’t hide. And everyone is going to have a different responses to it. Some ignore it, some will ask questions, and others…
Hypopituitarism affects many children with cerebral palsy. The condition causes poor growth, often delaying puberty. But there is good news for this community! A report was recently published called “Hypopituitarism…
What if you could prevent a rare disease from ever occurring? It sounds too good to be true. But researchers believe that psoriatic arthritis (PsA) may be preventable in patients…
Rare Diseases India (RDI) is a non-profit which was established in 2009. Its goal: to advocate for the citizens of India living with rare diseases, in addition to their counterparts…
On June 21st, Targeted Oncology posted the results from a phase 2 clinical trial for chronic lymphocytic leukemia (CLL). The trial examined the effects of combing two therapies--obinutuzumab and bendamustine.…
Communication in the medical field doesn’t happen as much as it should. But, when it does, great discoveries often occur. The thing is, science is complicated. Researchers have so much…
At 5 years old, Holton Jones was diagnosed with ROHHAD, or rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation. According to the Pensacola News Journal, his parents knew something…
Elizabeth couldn’t speak. She wasn’t expected to ever utter a word. She couldn’t read. She struggled to make trips outside of the house. Certain smells and loud sounds bothered her.…
Orkambi (ivacaftor / lumacaftor) ist die zweite Präzisionsmedizin, die für den Einsatz in Großbritannien von Menschen mit zystischer Fibrose zugelassen wird. Der Fang? Es wird derzeit nicht von der National…
Orkambi (ivacaftor / lumacaftor) es la segunda medicina de precisión con licencia para uso en el Reino Unido por personas con fibrosis quística. ¿La captura? Actualmente no es proporcionado por…
Imagine being told that your son probably wouldn’t live beyond his twenties when he was only six years old. Unfathomable. But that’s the news the Vella family received and had…
Aeglea BioTherapeutics recently provided an update concerning their clinical trial for arginase deficiency disorder. The four-word summary? They still need patients. Aeglea specializes in amino acid metabolism. Their focus is…
ChemoCentrx has been working to develop a drug to combat auto-antibody-associated vasculitis (AAV), C3 glomerulopathy (C3G), and atypical hemolytic uremic syndrome (aHUS). Its name- Avacopan. ChemoCentryx has always had a…
Treatment is expensive. The HealthWell Foundation gets it, and they’re here to help. Insurance companies don’t want to pay more than they have to for your medicine. It’s the nature…
On October 20th and 21st, the 2017 Rare Neuro-Immune Disorders Symposium (RNDS) will be held in Columbus, Ohio. The event is meant to bring together patients, families, researchers, and all…
Elly Mayday, model, is teaming up with Adam Lawrence, filmmaker, to spread awareness and support for those affected by ovarian cancer. Elly was diagnosed with stage 3 ovarian cancer just…
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.
