Phenylketonuria (PKU) is a rare disease which causes phenylalanine (an amino acid) to build up in the body, leading to serious health complications. Since amino acids are obtained through the…
For rare disease patients who face physical disabilities, even seemingly simple tasks can pose extreme difficulties. For instance, changing the channel on the television. Comcast Corp. previously created a voice…
The Study Juvenile idiopathic arthritis (JIA) is a rare disease that causes inflammation in the joints. It is of unknown origin and has no cure. Previous investigations in studies with…
Zogenix has just announced that they have not only finished enrollment in their Phase 3 investigation of Fintepla for Lennox-Gastaut Syndrome (LGS), but that the final patient has been randomized…
Eurordis-Rare Diseases Europe Eurordis-Rare Diseases Europe is an alliance of patient organizations which works to give rare patients a voice. It also strives to spread awareness of rare diseases to…
Heather White Heather White is a 41-year-old woman who was diagnosed with common variable immune deficiency (CVID) back in 2004 after fighting frequent bouts of pneumonia. Doctors believe that she…
This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…
Dinesh Khanna, a professor at the University of Michigan has worked heavily with the rare disease scleroderma. Michigan has its own Scleroderma Program, of which Khanna is the director. Through…
Happy 7-11 Day! We hope everyone's on their way to get their free slurpie today! For this week's editor's choice, we're highlighting four articles. First, we discuss kratom, the controversial…
According to a story from Pharmafield, The UK's National Institute for Health and Care Excellence (NICE) has recently approved a first in class gene silencing therapy for coverage on the…
According to a story from drugs.com, the biopharmaceutical company Mirum Pharmaceuticals recently announced that they have begun dosing the first patient in a phase 3 clinical trial. This clinical trial…
According to a publication at Markets Insider, the Food and Drug Administration (FDA) recently approved Soliris (eculizumab) as a treatment for neuromyelitis optica spectrum disorder. The approval marks the first…
Gaucher disease (GD) is a rare lysosomal storage disorder. It is caused by a deficiency in the glucocerebrosidase enzyme. In June, a new study was published in the International Journal of…
According to a story from PR Newswire, the drug selinexor (marketed as Xpovio), which was developed by Karyopharm Therapeutics, was recently approved by the US Food and Drug Administration (FDA)…
Growing up "Normal" Growing up, Steph Derham always thought she was normal. She says she has her parents to thank for that. Steph is incredibly grateful that her mom and…
Rare Disease Fund Many families affected by rare diseases cannot afford the necessary treatments. Due to the small population of people these diseases affect, exorbitant price tags are often placed…
According to a story from Market Screener, the biopharmaceutical company Theranexus and the Beyond Batten Disease Foundation (BBDF) have recently signed an agreement to work towards the continued development of…
According to a story from prnewswire.com, the biopharmaceutical company Aridis Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to the…
The FDA has approved a new therapy for chronic immune thrombocytopenia (ITP). This treatment is an oral therapy to be taken with food, created by Dova Pharmaceuticals. It is called…
Frankie Jr. Muretic Frankie Jr. Muretic lives in Mississauga in Ontario, Canada. One morning he woke up with a rash, causing his parents concern. They brought the five-year-old boy to…
According to a story from Japan Today, a recent ruling from a Japanese court has ordered the government of that country to pay damages to the tune of 370 million…
Happy Fourth of July Season! Today, we're highlighting stories about research and social media, robots in caregiving, sisters carrying each others' babies, and AI advancing glioblastoma research. After this past…
Dicerna Pharmaceuticals has just announced that they have submitted a Clinical Trial Authorization application for their investigational therapy DCR-A1AT. This application has been submitted to the Swedish Medical Products Agency…
According to a story from BioPortfolio, the biopharmaceutical company X4 Pharmaceuticals, Inc. has recently initiated a phase 3 clinical trial that is testing its current lead product candidate mavorixafor. This…
According to a story from BioPortfolio, the drug company Pfizer has recently presented data from its phase 1b clinical trial that tested the company's experimental gene therapy PF-06939926, which is…
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