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Once upon a time there were three young brothers, and SPOILER ALERT- this part of the story, unfortunately, does not have a happy ending. Each brother suffered: Each had chronic…
1. On Thursday, June 1st at 12 pm EST, 11 am central, there will be a seminar by the Amyloidosis Foundation to better understand this complex disease. It will be narrated by two physicians from…
A special session on CGD will be presented at the 2017 IDF National conference. CGD is a primary immunodeficieny disease and is characterized by skin infections, both fungal and bacterial,…
A rare disease that is not so rare: Hormonal causes of high blood pressure have been considered rare but “can be as high as 50% among children and 30% among…
Remember when PW reported on the Newborn Screening and Gene therapy for the Immune Deficiency SCID? Well guess what our friends at the Immune Deficiency Foundation are doing? The IDF…
Do you have VASCULITIS? Associated with: Behcet’s Disease, Churg Strauss Syndrome, Cryoglobulinemia, Giant Cell Arteritis (Temporal Arteritis), Granulomatosis with Polyangiitis (formerly Wegener’s Granulomatosis), Hpersensitivity Vasculitis (Leukocytoclastic), IgA Vasculitis (Henoch-Schönlein Purpura), Kawasaki…
Are you an adult who has been diagnosed with Dermatomyositis (DM) or probable DM? There is a new Phase II study currently recruiting that you may want to discuss with…
2017 Muscular Dystrophy Association Scientific Conference was a beacon of light! Researchers from academic medical centers, pharmaceutical companies, children’s’ medical centers, biotech, engineering, and veterinary medicine came together to share…
Baby Isabella (pictured above) is in serious need of a hospital transfer. Her condition Gastroschisis, is rare. She needs a facility with people who have the most experience caring for…
It is hard to study rare diseases because there are so few people with them. Patients are scattered all over the world. Many are children. Many cannot travel. How to make…
GSK and the National Institute of Allergy and Infectious Diseases have some good news released on Thanksgivng Day and its name is Nucala. Vasculitis is a tough and not fully…
The physicians who left me undiagnosed said "we" missed the brain tumor. WE?! YOU, the 10 or 12 doctors I saw, missed that I had acromegaly. The doctor who finally…
Fabry Disease is the number number two Lysosomal Storage disease in terms of numbers affected. It is an X-linked disease, with males passing on the mutated allele to all of their…
Gastroschisis is a birth defect where the intestines and sometimes other abdominal organs such as the stomach or liver are outside the body, rather than inside. This used to come as…
Current treatments for cervical dystonia provide inadequate relief to many. Dystonia twists people and leads to strange postures, involuntary movements and pain. It affects men, women and children. Check out more…
Sociologist Georg Simmel, calls Gratitude the “ moral memory of mankind”. But researchers tell us that gratitude is more than a moral or ethical acknowledgement of help of some kind.…
Hunter Syndrome, is a heartbreaking twist of genetic fate, and we celebrate that the FDA has granted Rare Pediatric Disease Designation to REGENXBIO's RGX-121 Gene Therapy. Hunter Syndrome causes seemingly…
Jana and Sara are 32 year old twins, born and raised in North Dakota. We had the pleasure of speaking with both of these precious young ladies about their rare…
Still not feeling well? You are not alone! The new, and validated survey instrument on quality of life for patients being treated for acromegaly, developed by Dr. Maria Fleseriu and…
Just weeks after the FDA required more data prior to approving an oral form of Octreotide, proposed by Chiasma Pharmaceuticals, a British pharmaceutical and device company, Glide (not the dental…
Have a rare disease? Have you ever been to a national patient conference? These are invaluable opportunities to connect with others who share your challenges and concerns! Imagine being in…
“ Many describe the symptoms of HD as having ALS, Parkinson’s and Alzheimer’s – simultaneously.” - The Huntington’s Disease Society of America States For almost 50 years research into the cause and…
A mother of three in Scotland had experienced dystonia symptoms since childhood, and these symptoms got progressively worse over the years. Like so many with a rare condition it took…
The HAE community cheered in July of 2015 when another new therapy was approved by the FDA to treat acute HAE attacks. Now, the pharma company has another reason to cheer. The…
Life just got a little easier for parents of young kids with Tyrosinemia Type I (HT-1) Raising a child with Tyrosinemia requires constant, attention to diet, a special formula, and…
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