Second Annual International Congress on Advanced Treatments in Rare Diseases The first International Congress on Advanced Treatments in Rare Diseases, otherwise known as RARE2018, was held in London, England. Now,…
Tommy Salisbury is eighteen years old. Unlike many teenagers, he is making big moves in the fundraising community and working hard to be a leader in efforts to find…
What is Pulmonary Arterial Hypertension? Pulmonary arterial hypertension (PAH) is a rare form of high blood pressure. Normal blood pressure is around 14 mm HG at rest. Someone with PAH…
What is Distal Renal Tubular Acidosis? Distal renal tubular acidosis (dRTA) is a rare condition which affects approximately 1 in every 100,000 people worldwide. It is caused by a build up…
Rare Disease Legislative Advocates' Legislative Conference Part of Rare Disease Week on Capitol Hill 2019 All of the events for Rare Disease Week are free to patients, advocates, and caregivers.…
Cocktail Reception and Rare Disease Documentary Part of Rare Disease Week on Capitol Hill 2019 All of the events for Rare Disease Week are free to patients, advocates, and caregivers.…
According to a story from Food Safety News, an illness known as chronic wasting disease has been known to affect members of the deer family in the US since the…
According to a story from Healio, wearable technology that is meant to help users monitor their health and fitness have continued to grow in popularity in recent years. Most people…
According to a story from PR Newswire, the specialty pharmaceutical company Mallinckrodt plc recently announced the release of interim data from the company's Phase 3 clinical trial. This trial is…
According to a story from Oncology News, a team of researchers affiliated with UNSW Sydney have made a discovery that could have major implications for the rare cancer neuroblastoma, a…
The typical treatment for Fabry disease is Fabrazyme which is an enzyme replacement therapy (ERT). This therapy must be administered to the patient every two weeks. A new ERT created by…
What is the Patient Advocate Foundation? The Patient Advocate Foundation (PAF) is a non-profit, formed in 1994 that has one primary aim- eliminate the barriers to treatment too many patients…
Cystic Fibrosis Cystic fibrosis (CF) is a rare condition that causes mucus to build up in the lungs and the digestive system. It results in chronic lung infections, lung inflammation, and…
Langerhans Cell Histiocystosis Langerhans Cell Histiocytosis (LCH) Is a rare form of cancer caused by an excessive amount of Langerhan cells in the body. These cells form granulomas which can have a…
Fetal Surgery for Spina Bifida Spina bifida is a rare condition which results in improper spine formation during embryonic development. A gap occurs in the vertebrae causing nerve damage in varying…
Hydrocephalus Hydrocephalus is a rare disease which causes a build up of fluid in the ventricles of the brain. The continuous flow of this fluid is how the body prevents the…
Happy Wednesday! Today, we're highlighting an article about gene therapy for MPS, followed by a story about a singer with Achalasia. After that, we have an article about app for…
According to a story from BioSpace, the biopharmaceutical company Modis Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted Breakthough Therapy designation for the company's experimental…
A grandmother of four in Maryland has recently become the first cancer patient to complete the experimental CAR-T cell procedure at Georgetown University Hospital. Daisy Diggs 67, who was…
What is Precision Medicine? In essence, precision medicine refers to the idea that in order to establish the best treatment plan for a patient, we must look at them as…
According to a story from prnewswire.com, the biopharmaceutical company Neurotech Pharmaceuticals recently announced that the company had earned Fast Track designation from the US Food and Drug Administration (FDA). This…
The first step in treating rare diseases is accurately diagnosing them. Unfortunately far too many people go years without knowing what is going wrong in their body. Doctors are trained…
Ocugen Pharmaceuticals has just received their first ever orphan drug designation by the FDA for a gene therapy treatment. It's called OCU400 and it's being investigated as a treatment for…
Cystic Fibrosis Cystic fibrosis (CF) is a rare disease caused by the lack (or malfunctioning) of the cystic fibrosis transmembrane conductance regulator (CTFR) protein. This occurs when children inherit a mutated…
Neutropenia Neutropenia is a rare condition that affects approximately 1 in every 200,000 newborns. It results in a reduced number of neutrophils in the body. Neutrophils are white blood cells,…
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