According to a publication from ET Healthworld, some 21 rare disease patients in India's northern state of Uttar Pradesh are still awaiting governmental financial assistance to which they are legally…
Cleveland Browns wide receiver Jarvis Landry has been affected personally by CF; he lost his high-school sweetheart Mya Zimmer in July of 2015 after her long battle with the rare…
According to a story from BioPortfolio, the biopharmaceutical company Genkyotex has recently announced that the US Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application.…
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According to a report recently published in the Inter Press Service, since the advent of multidrug therapy in 1982, over sixteen million people have been cured of Hansen’s disease,…
The Dog Days Are Back Is it hot or what!? Whether you're trying to catch a tan or stay cool, we have a fresh dose of weekly content in Editor's…
According to a press release from LifeMax Laboratories, Inc., the Food and Drug Administration (FDA) has granted the Company's experimental Netherton syndrome drug LM-030 (licensed from Novartis) Rare Pediatric Disease…
Femtech Femtech refers to any type of technology that is centered on women's healthcare needs. This includes wearable breast pumps, apps to increase pelvic floor strength, menstruation apps, ovulation apps,…
According to a story from bloomberg.com, the drug development company Arrowhead Pharmaceuticals, Inc. recently announced that its experimental product candidate AR0-ANG3 has earned Orphan Drug designation from the US Food…
According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This…
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…
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