A new genetic test for keratoconus as well as other types of corneal dystrophy was released late last year. The test was created by Avellino and can identify biomarkers of disease. It also looks…
The CureGRIN Foundation has recently announced that its annual conference will be held virtually this year as a result of the ongoing coronavirus/COVID-19 pandemic. This year's event will be held…
by Lauren Taylor from In The Cloud Copy Nearly all babies born in the United States today are tested for a certain panel of genetic disorders within 48 hours of…
by Natalie Homan from In The Cloud Copy In July of this year, doctors in Switzerland started using a new software program that will help them and many others more…
by Danielle Bradshaw from In The Cloud Copy Elaina Pechota loves to dance. Back in 2018, however, the teen had a fall during one of her classes, and her entire…
Claire Brinkley Claire Brinkley's daughter Eleanor is diagnosed with progressive familial intrahepatic cholestasis (PFIC) which is a rare disease affecting the liver. As a newborn, she appeared perfectly normal. But at…
According to a story from Charcot-Marie-Tooth News, the nonprofit organization CMT Australia, which focuses on serving the Charcot-Marie-Tooth disease patient community in Australia, has recently acquired a grant to the…
According to a story from BioSpace, the biopharmaceutical company Cerecor Inc., has recently announced that it has restarted operations of its phase 1b clinical trial. This trial is evaluating the…
According to a story from eminetra.com, 37 year old sarcoidosis patient Sonja Nixon Frazier sprung into action when she was working at Taco Bell. She along with two workers knew…
According to a story from BioSpace, a case study of a 33 year old man has confirmed that it is possible for humans to get infected with the virus responsible…
According to a story from england.nhs.uk, a recent agreement struck between the UK's NHS and Vertex Pharmaceuticals will herald a major improvement for the many of the country's cystic fibrosis…
According to a story from ScienceBlog, a comprehensive study which focused on children diagnosed with coronavirus/COVID-19 produced some results that surprised scientists and suggested that children could play a greater…
by Lauren Taylor from In The Cloud Copy Duchenne muscular dystrophy (DMD) is a rare genetic condition that ultimately leads to the wasting away of muscles over time. The condition…
Female mammals have XX chromosomes and males have XY. But scientists have never understood how females decide which of their two X's should be active in each of its cells.…
Dr. Harvey Moldofsky Harvey Moldofsky is a professor emeritus at the University of Toronto. His area of focus is SARS, and since the onset of COVID-19, he has been fully…
This past week, biopharmaceutical company Mustang Bio announced that their gene therapy candidate MB-107 received Rare Pediatric Disease Designation. The gene therapy candidate is designed to treat X-linked severe…
To capture the essence of palliative care, the World Health Organization defines it as the prevention and relief of suffering associated with a life-threatening illness. An article in Parkinson’s…
Ross Douthat, a writer for the New York Times, was first diagnosed with Lyme disease and a Bartonella infection in 2015 from a deer tick bite. The effects have lingered…
A press release issued recently by Regeneron Pharmaceuticals heralded the acceptance of a Biologics License Application by the FDA for Priority Review of its investigational drug, evinacumab. The Priority…
Ascendis Pharma has just announced that their investigative therapy for achondroplasia called TransCon CNP has just received Orphan Drug Designation from the European Commission. This same designation has already been…
Zacari was born with a rare neuromuscular disorder called arthrogryposis multiplex congenita. Children with the disease are unable to straighten or bend their legs or arms. Yet, according to…
Do you want to make a difference in patient advocacy and rare disease education? Well, here's your chance. Every year, the Dystonia Medical Research Foundation (DMRF) holds a local…
The Vestibular Disorders Association has announced its first ever Virtual Vestibular Conference. The virtual event is scheduled from September 14-18 2020 and is being held as part of Balance Awareness…
This week, Regeneron Pharmaceuticals announced that their Biologics License Application (BLA) for evanicumab, a treatment for homozygous familial hypercholesterolemia (HoFH), was granted Priority Review status by the FDA. The…
According to a story from curetoday.com, patients living with mantle cell lymphoma, a rare form of blood cancer that makes up only around six percent of diagnosed non-Hodgkin lymphoma cases…
© Copyright 2024 Patient Worthy
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.
