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…
The U.S. Food and Drug Administration has granted the experimental drug A4250 rare paediatric disease designation for the treatment of progressive familial intrahepatic cholestasis. The full article can be found…
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…
I've known some wonderful fathers throughout my life. My own father was a gem. My siblings agree. However, it was not until I became immersed in the rare disease world…
Newron Pharmaceuticals S.p.A (Newron) is a biopharmaceutical company specializing in developing inventive therapeutic options. They focus primarily on conditions related to the nervous system. According to bioportfolio.com, Newron announced on…
According to BioPortfolio, there was recently an announcement about preclinical data for SYNB1618, which is a Synthetic Biotic treatment that is being assessed right now in a Phase 1/2a clinical trial in…
Many people are familiar with Carl Zimmer as a science columnist from The New York Times. His columns and research dug deeply into the field of genetic research and gene…
According to EurekAlert!, a study at Virginia Commonwealth University has potentially pinpointed the so called "Achilles heal" of a certain type of deadly brain cancer: glioblastoma multiforme. Glioblastoma, or glioblastoma…
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…
According to Chipley Bugle, the University of Florida Health Center is joining the Pulmonary Fibrosis Foundation’s Care Center Network. UF joins 60 other centers in the world with high level knowledge…
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…
Click to read parts one and two of Joanie's story. Since 2012, when I left my job as an educator, I have tried many different modes of medical therapies. I'm…
According to WBUR, treatments like CAR-T and CRISPR are on the cutting edge. They’re important to keep an eye on as the future of medical technology and treatment. They’re not…
According to Globe Newswire, the United States Food and Drug Administration (US FDA) designated a new orphan drug for the treatment of multiple myeloma on May 9th, 2018. The drug…
I am a caregiver mom to my two special needs, young adult, rare disease kids. I am also a recent widow; previously, I was a caregiver wife to my very…
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,…
Lawmakers in the U.S. state of North Carolina have expanded the state’s newborn screening testing to include three more conditions. These are Pompe disease, Mucopolysaccharidosis type 1 (MPS 1), and…
According to Charcot-Marie-Tooth News, a Chinese family of five who all have the same type of Charcot-Marie-Tooth (CMT) disease share something else in common as well. Not only do they…
Before I start, I want to remind everyone that I am not your doctor or a halfway adequate replacement for your doctor. If you find that you're fainting regularly-- whether…
To read part one of Joanie's story, click here. In 2014, I developed excruciating back pain and eventually the doctors found I had two spinal cord tumors. One was on…
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…
There are many mysteries still contained in human biology. Circadian clocks and DNA repair seem to share an interaction that may be one of them. Furthermore, based on research from…
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