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…
Hemophilia can affect anyone. Let’s make a few things clear: Women are carriers of hemophilia and pass the gene, but men determine the child’s gender. It’s not all on you,…
Medical Research Network (MRN) is an organisation that specialises in the conduct of clinical trial visits in the patient’s home and supporting clinical trial sites with nursing resources. MRN aims…
Mallory and Dave Holmes were excited. Like many couples, they were eagerly awaiting the birth of their first child. After 20 weeks of pregnancy, however, the couple received news that…
To the patient: Accepting your diagnosis doesn’t mean you can’t be mad about it. It simply means you understand that the reality of your life has changed and you know…
According to a story from the National Institutes of Health, a recent study has revealed that the absence of a specific protein in the cells lining the esophagus could be…
According to a story from PLOS Medicine, most people will probably tell you that the anti-vaccine movement in the United States is a total failure. While the vast majority of…
According to PRNewswire, Express Scripts announced a new program to benefit people living with rare diseases on May 31st. The program, called Second Opinion is part of the company’s larger…
Happy Friday everyone! This was a week with a highlight on research: we cover the state of ME/CFS information at the moment, an exciting treatment approval for people with ulcerative…
According to a story from Rare Disease Report, Nichol Kirby, who was diagnosed with acute intermittent porphyria in 2015, faced an arduous path to her diagnosis. Her constant pain, which…
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