Achondroplasia is one of those illnesses that you can’t hide. And everyone is going to have a different responses to it. Some ignore it, some will ask questions, and others…
Hypopituitarism affects many children with cerebral palsy. The condition causes poor growth, often delaying puberty. But there is good news for this community! A report was recently published called “Hypopituitarism…
What if you could prevent a rare disease from ever occurring? It sounds too good to be true. But researchers believe that psoriatic arthritis (PsA) may be preventable in patients…
Few things are as frustrating and complex as the health care system in the US. Even the current President admitted to that fact. So, like many people in the rare disease…
In April of this year, Argos Therapeutics reported interim results from its Phase 3 ADAPT clinical trial of rocapuldencel-T in combination with the normal standard-of-care for the treatment of newly…
The week before last, innovative biopharmaceutical company DelMar, presented data at the Pediatric Neuro-Oncology Basic and Translational Research Conference from a clinical research with VAL-083 in chemo-resistant glioblastoma patients. Glioblastoma is…
The biopharmaceutical research and development company CytRx Corporation, announced an exciting new update early this month involving the company’s work with aldoxorubicin, a new drug for treatment in soft tissue sarcomas.…
Rare Diseases India (RDI) is a non-profit which was established in 2009. Its goal: to advocate for the citizens of India living with rare diseases, in addition to their counterparts…
Cystic Fibrosis… For a mother, those words are disheartening, confusing, and so, so frightening. For a child, those words mean countless daily medications, extensive hospital visits, and a shortened life…
I will freely admit: I am terrible, terrible, TERRIBLE about drinking enough water each day. Do I know it’s important? Of course I do! Do I recognize the health benefits?…
Albireo Pharma is in the midst of preparing for an exciting Phase 3 clinical trial to test A4250 for the treatment of progressive familial intrahepatic cholestasis (PFIC). PFIC is a…
TGIF Patient Worthians! This week we have promising results on promising results. First up is news on a Dravet syndrome treatment. Second is a Lupus Nephritis drug in the works! Third…
It is every woman's dream. After nine months of waiting, anticipating, dreaming, and expectancy, Jillian Johnson’s first baby was finally in her hands. Her new bundle of joy, Landon, was…
On June 21st, Targeted Oncology posted the results from a phase 2 clinical trial for chronic lymphocytic leukemia (CLL). The trial examined the effects of combing two therapies--obinutuzumab and bendamustine.…
Onxeo has recently received the 10th unanimous recommendation from the Data Safety Monitoring Board to continue with the Phase 3 study (coined ReLive) of Livatag®. Livatag® is designed for use…
Many sisters may play pranks or bicker with each other often. According to ABC 11 News, Twelve-year-old Natalia and fourteen-year-old Nyasia Yarrell are no different. One minute they are mad at…
You're happily tucking your toddler in for her afternoon nap when she starts to panic. Where's her favorite blankie and stuffed penguin? A peaceful nap won't occur without it! With…
Xynomic Pharma, a United States-based oncology research and development company, announced at the end of last month that it had entered into a partnership with the University of California San…
The line between genius and madness is often closer than people realize. Don’t tell Dr. Sergio Canavero that he can’t help his patient with spinal muscular atrophy (SMA). How many…
Communication in the medical field doesn’t happen as much as it should. But, when it does, great discoveries often occur. The thing is, science is complicated. Researchers have so much…
At 5 years old, Holton Jones was diagnosed with ROHHAD, or rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation. According to the Pensacola News Journal, his parents knew something…
The Danish biotechnology company, Orphazyme, announced in early May of this year that enrollment was completed for its lead study program for Niemann-Pick type C disease. Niemann-Pick disease is a…
Support for Ofev® just keeps coming! Ofev® (nintedanib) was first approved by the U.S. FDA in 2014 for the treatment of idiopathic pulmonary fibrosis (IPF) after clinical trials, especially the INPULSIS…
Results from the Phase 3 CASTOR trial from Janssen Pharmaceutical Companies were recently released, finding that Darzalex (daratumumab) was extremely effective at reducing the risk of multiple myeloma progression or…
A new public cord blood donation center recently opened up in New York. This is the second of this type of facility to open in the state. In the past,…
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