Spinal muscular atrophy (SMA) occurs when the immune system attacks motor neurons, resulting in the muscles surrounding the spinal cord atrophying (degenerating). SMA is an inherited disorder that disables the…
The Pulmonary Fibrosis Warriors (PF Warriors) and monARC Bionetworks teamed up today to host a Facebook Live video, sharing important information about how patients can become more involved in research…
The Associated Professional Sleep Societies held their 31st annual meeting in Boston June 3rd through 7th. You can read more about Sleep 2017 by clicking here. Among other discussions was cataplexy.…
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…
Imagine your father has just suffered a heart attack and must undergo open-heart surgery in order to repair the damage... Picture your baby niece or nephew was born with a…
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…
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…
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