In the US, the rare condition glomerulonephritis is the third leading cause of the last stage of chronic kidney disease (CKD). A clinical trial sponsored by the National Institutes of…
Contagion Live (Infectious Diseases Today) recently discussed a journal article on the prevalence of atopic dermatitis. The simple summary? More research is needed to uncover just how common this condition is. BUT…
During this year’s International Workshop on chronic lymphocytic leukemia (CLL), Shih-Shih Chen, PhD, was recognized for her innovative research. Her research could mean a cure is around the corner for CLL.…
Annually, 13 out of 100,000 people are diagnosed with trigeminal neuralgia. Trigeminal neuralgia is described as a sharp and intense pain that can occur in the face or mouth. The…
By examining a zebrafish, scientists have found the gene that affects the maintenance of cone photoreceptors and visual functions in the eye. It might be the knowledge that’s been needed…
The U.S. Food and Drug Administration (FDA) approved Biogen’s SPINRAZA™ (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only…
Too often rare diseases such as tardive dyskinesia (TD) are treated with medications and therapies that were made for other illnesses. This occurs pre-diagnosis, by physicians who have never seen…
According to the Daily Mail, Clare Fray has alpha-1 antitrypsin deficiency. But for years she thought her symptoms were just a part of normal life. She suffered from numerous chest…
It’s amazing how much we take for granted. Eating. Seeing. Walking. There are a million and one things that can go wrong with the human body, and until one of…
A phase 3 clinical trial has produced positive results for cutaneous T-cell lymphoma (CTCL) patients. The drug tested is called brentuximab vedotin (Adcetris). Specifically, the drug was created to treat…
Living with PBC: Learning Together For patients, family, healthcare professionals, and so many more! FEATURING: Certified PBC Nurse Educator, Jennifer Blood, and a Patient Ambassador, Connie Call 1-844-514-6294 to register and…
Dear Primary Care Clinics, Rare Disease Warriors Are Not Pariahs; We Are Your Patients. -From a SPS Fighter As a rare neuromuscular disease patient, who fought 21 years for a…
According to CBC, Marie-Eve Chainey was on dialysis six out of seven days a week. For 15 years, this was her life. It was a necessary feat to control her…
Case Western Reserve University researchers have recently developed gene-carrying nanoparticles that are able to bring healthy genes to those negatively affected by Leber congenital amaurosis (LCA), a disease that causes…
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…
Have you ever just felt so desperate for something that you jump into a decision, without thinking it through completely or weighing the possible repercussions of your decision? It had…
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…
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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…
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