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For many rare diseases, gene therapy, not traditional therapeutics, is perhaps the only means to significant relief and cure. However, the path to gene therapy has been strewn with obstacles…
On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983). The Draft Guidance considers…
Heidi Jo Price, beautiful and brave, has jumped out of airplanes with a pack on her back as a member of the 173 Airborne Brigade Combat Team. What brought this…
The EveryLife Foundation for Rare Diseases hosted a panel discussion on Right to Try Proposals, as part of their 2018 Legislative Program for Rare Disease Week on Capitol Hill. What…
United in Progress is the tag line for a unique collaborative effort to speed progress in identifying, understanding and treating hemophilia: My Life Our Future. Increasingly in the area of rare…
It is absolutely and unequivocally clear : “The PKU diet must include a medical product, usually consumed as a beverage. There are several brands available that are nutritionally suitable for…
In 1972 William S. Sly, first identified a rare, progressive and devastating condition, which came to be known as Sly Syndrome, now known as MPS VII. This syndrome affects almost…
It is Patient Worthy’s pleasure to report on the first International Acromegaly conference which brought together acromegaly support groups from East and West Canada,- The Vancouver and Atlantic Acromegaly support…
The International Myotonic Dystrophy Consortium was held in San Francisco last month and while Patient Worthy was not there in person we were fortunate to have a family reporting from the…
On September 28th, 2017 at 9 PM EST, a new online, mindfulness-based, stress reduction course will be given by none other than the extraordinary Julie Desch, MD. She is a physician…
The Indiana Hemophilia & Thrombosis Center in Indianapolis has long been a leader in innovative patient care. They teamed up with MicroHealth, a company founded by hemophilia patient Aaron Craig…
Patient Worthy attended the National Hemophilia Foundation's 69th Annual meeting in Chicago and we are thrilled to report on some of the exciting new developments for patients with bleeding disorders.…
Patient Worthy’s very own contributor Iva Rauh, with a tiny nudge from Patient Worthy, attended the International Pemphigus Pemphigoid Foundation’s Conference in Lubeck, Germany. Who better to learn about the latest developments than…
Patient Worthy attended the NIH‘s National Center for Advancing Translational Sciences NCATS meeting and in a word: WOW! Start with the patient- the end user, the source of the aberrant…
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…
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