Tags Posts tagged with "rare disease"

rare disease

After the 10th International Workshop on Waldenstrom’s Macroglobulinemia for researchers and clinicians, the IWWM10 International Doctor- Patient Forum will be held in the same...

Put Europe on your calendar for 2018! Usher patients, researchers and family members welcome to the two-day Symposium in July. For more information, contact ush2018@usher-syndrome.org

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...

According to a story from BioPortfolio, the gene therapy company Nightstar Therapeutics recently released an announcement that the FDA issued Regenerative Medicine Advanced Therapy...

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...

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...

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,...

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...

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...

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...

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...

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...

A Nigerian mother, Hafsat Aliyu has penned a moving tribute to her beautiful daughter, Zainab, who died of Hodgkin lymphoma, a type of blood...

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...

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...

Mallory and Dave Holmes were excited. Like many couples, they were eagerly awaiting the birth of their first child. After 20 weeks of pregnancy,...

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...

According to a story from the National Institutes of Health, a recent study has revealed that the absence of a specific protein in the...

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...

According to PRNewswire, Express Scripts announced a new program to benefit people living with rare diseases on May 31st. The program, called Second Opinion...

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...

According to a story from Rare Disease Report, Nichol Kirby, who was diagnosed with acute intermittent porphyria in 2015, faced an arduous path to...

According to a story from pm360online.com, the clinical stage corporation Epizyme recently released detailed results from the Phase 2 trial of its leading investigational...

According to a story from empr.com, a recent study revealed that gene therapy could allow patients with beta thalessemia to stop having to get...

Patient Worthy is proud to announce some new features to our website! Thanks to your valuable feedback, we have now enabled the personalization of your...