A pie to the face?! Yes, you heard right! Saturday, June 30th is Arthrogryposis Multiplex Congenita (AMC) awareness day! It is our duty here at Patient Worthy to shine a light…
Andrea Jenkyns is a member of the British Parliament and new mother. She was elected as the Conservative Member of Parliament for Morley and Outwood at the 2015 general election.…
According to a story from uk.news.yahoo.com, when Billy Caldwell, age 12, and his mother returned from traveling to Canada, the Home Office initially confiscated a substance that had been the…
According to a story from Human Rights Watch, stories of preventable fatalities due to medical emergencies in immigrant detention centers in the U.S. have begun to emerge. In many cases,…
According to a story from BioPortfolio, the biotechnology company Vertex Pharamceuticals, Inc. recently announced that the long term use of its innovative cystic fibrosis treatment Orkambi will be covered in…
According to medicalxpress.com, Hepatocellular carcinoma is the third leading cause of cancer deaths in the world. It is also the most common form of liver cancer. New research from a…
Arrowhead Pharmaceuticals, Inc. have announced that they have completed patient enrollment for a Phase 1 clinical trial investigating ARO-AAT, an investigational therapy for the treatment of a genetic liver disease…
According to a story from Business Wire, the company Alexion Pharmaceuticals recently announced that it has submitted a Biologics License Application (BLA) to the FDA for the approval of one…
According to a story from CheckOrphan, Express Scripts has prepared a Rare Conditions Care Value (RCCV) program. This program is designed to help make the diagnosis and treatment process for…
Happy start-of-summer everyone! We're kicking off the summer with a wrap up on rare disease news this week. We have an article about a systemic mastocytosis study, and a clinical…
According to a story from apnews.com, Mike Coble, a senior at Byron High School in southeastern Minnesota, was recently diagnosed with Guillain-Barre syndrome. Mike is on three different sports teams…
Data from a Phase II clinical trial of the experimental treatment reldesemtiv for the treatment of spinal muscular atrophy has been announced. The drug has shown positive effects in the…
According to a story from UK Healthcare, a team of researchers and scientists at UK Healthcare is preparing to start a clinical trial that will test a new therapy aimed…
The experimental gene therapy LentiGlobin for the treatment of severe sickle cell disease is being investigated in an ongoing Phase 1 clinical trial. Bluebird Bio, the company developing the therapy,…
Saturday June 23rd is Dravet syndrome awareness day! Dravet syndrome is a rare genetic dysfunction of the brain (epileptic encephalopathy). It begins in the first year of life in an otherwise…
According to a story from BioPortfolio, the gene therapy company Nightstar Therapeutics recently released an announcement that the FDA issued Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational product…
According to a story from pm360online.com, the drug development company ProQR Therapeutics N.V. will be partnering with EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF) in order to…
According to a story from CBS News, Clay Emerson, from New Jersey, is hoping to use Father's Day in order to raise awareness about his three year old daughter's rare…
Finding the right doctor for a patient can often be a long and personal process. It’s not just a matter of effectiveness or efficiency, but a matter of comfort and…
Happy Father's Day weekend! While Father's Day can be complicated in a rare family, rare parents deserve a round of applause. This week we have a Father's Day tribute from…
According to a story from pm360online.com, the speciality pharmaceutical company Chiesi USA and the biotech company Holostem Terapie Avanzate S.r.l. of Italy recently announced that the U.S. FDA recently granted orphan…
PatientWorthy will be offering a pair of tickets to the Alagille Syndrome Alliance's (ALGSA) First Annual Gala. The tickets will be available for patients or caregivers in the San Diego…
According to a story from med.miami.edu, a team of researchers working at the Sylvester Comprehensive Cancer Center, which is part of the University of Miami School of Medicine, made a…
The below article was originally published on June 14th, 2018 in Miracle Flights Newsletter, The Co-Pilot, and is being republished here with permission. Rebecca is big sister to 10-year-old Amanda…
A Nigerian mother, Hafsat Aliyu has penned a moving tribute to her beautiful daughter, Zainab, who died of Hodgkin lymphoma, a type of blood cancer, on May 7, 2015. She…
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