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…
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…
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…
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