It's not unusual for a disease like psoriatic arthritis to come with the risk of having other conditions, or a comorbidity. More than you might think, people with psoriatic arthritis also…
Early this month, Novocure announced that the first patient was enrolled in the RTOG Foundation's Phase 2 pilot trial for recurrent glioblastoma. This trial is aimed to test the safety and…
According to an awesome story in DelRay Newspaper, Kelsey Diamantis had always dreamed of going to the Kentucky Derby at Churchill Downs to watch the horse race. While thinking about…
Biohaven Pharmaceutical Holding Company Ltd. announced at the end of last month that the company has completed enrollment in its Phase 3 clinical trial of trigriluzole in spinocerebellar ataxia (SCA)…
In April, Prometic Life Sciences Inc. announced it would be submitting a Biologics License Application to the FDA for treatment of hypoplasminogenemia. The company's therapy has already been listed with Orphan…
The goal of the Alpha-1 Foundation is ultimately to find a cure for Alpha-1 Antitrypsin Deficiency (A1AD). But along the way, they are also a resource for patients, families, and…
Gastroparesis causes paralysis of the stomach. It basically means the stomach doesn’t understand its job, and can’t digest food properly. This causes extreme discomfort (to say the least) for gastroparesis…
Andrew Belliveau started the Pie Face Challenge in July of 2016 in order to raise awareness for gastroparesis and stimulate accelerated research for the disease. He took inspiration from the…
The Governor of Vermont made an official written proclamation March 3, 2017. He declared March as Bleeding Disorders Awareness Month, the state's first official observation of the cause. With this…
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…
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…
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…
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…
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…
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