Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
48 years of marriage, 2 children, 2 nephews, 7 grandchildren, and 3 great-nieces. It is clear that Lorrie and Stewart Altman, over their time together, have created a beautiful life.…
Stoke Therapeutics and Acadia Pharmaceuticals made a joint announcement this week through Business Wire of their collaboration in the development of RNA-based medicines to potentially treat rare neurodevelopmental disorders of…
CURE® recently conducted an interview with von Hippel-Lindau (VHL) patient Joseph Heisler and his mother, Marirene. Joseph’s father was diagnosed thirty years ago with the genetic (inherited) disease. Shortly thereafter…
According to a relatively recent news release from biopharmaceutical company Maze Therapeutics, the company has begun dosing healthy volunteers in a Phase 1 clinical trial. During the trial, researchers are…
Dr. Douglas Marchuk, professor of genetics at Duke, has spent years studying the genetics of cardiovascular disease. According to a recent article in the Atlantic, his research at the University…
One of the burdens that rare disease patients often face falls into the financial category, and that's treatments that come at a very high cost. One patient, a young girl…
Unfortunately, a pancreatic cancer diagnosis also comes with a poor prognosis. Since many people do not receive a diagnosis until later stages of the cancer, the 5-year survival rate is…
What prognostic tools can be used to assess patients with idiopathic pulmonary fibrosis (IPF)? How can researchers use these tools to determine the efficacy of therapies during clinical trials, or…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Mark Hoppus has been working in the music industry for three decades; his band, Blink 182, was formed back in 1992. He has a strong love for writing, performing, and…
When Nikolas and Ryan Michaud think of their little sister Jillian, they think of the joy she brings to their life. Nikolas says that Jillian's hugs are unmatched, maybe the…
In a news release from February 16, 2022, clinical-stage biopharmaceutical company AN2 Therapeutics, Inc. ("AN2") shared that its therapy epetraborole was granted Orphan Drug designation for the treatment of non-tuberculous…
When Sam Anderson was thirteen years old, he was diagnosed with familial adenomatous polyposis (FAP), a rare inherited disorder. As he grappled with his diagnosis and symptoms, Sam also…
According to a recent article, a pilot study shows that around 30% of patients with wet age-related macular degeneration might have the ability to stop their eye injection therapy…
On February 16, 2022, Healio shared that CT103A, a therapy for adult patients with relapsed or refractory (R/R) multiple myeloma (MM), had received Orphan Drug designation from the United States…
Continued From Part One Case #2 The second case involved a sixteen-year-old male in the ICU unit with suspected bacterial hypoxic respiratory failure associated with viral pneumonia. The teen’s history…
According to a recent article, Dr. Jyoti K. Jaiswal shared his research pertaining to gene therapies to be used to treat limb-girdle muscular dystrophy 2B (LGMD2B). Limb-Girdle Muscular Dystrophy (LGMD)…
According to a recent article, trametinib has proven to be successful in treating a rare ovarian cancer subtype, which is a major advancement in treating those with this rare ovarian…
Despite undergoing clinical development, many drugs never receive approval from the FDA. Recently, this happened to a treatment for growth hormone deficiency (GHD). The medication, titled somatrogon and owned by…
CFTR gene mutations cause cystic fibrosis, a rare genetic condition which causes progressive digestive and respiratory system damage. Yet it can be extremely difficult to treat for those with “stop”…
Claire Barrow is a 14 year old CEO, entrepreneur, rare disease patient, and advocate for the rare disease community. She is the founder and CEO of an app called RareGuru which…
An estimated 1 in every 200,000 Americans has lymphangioleiomyomatosis (LAM), a rare lung disease that disproportionately affects females. To this day, researchers are still learning new information about this condition.…
The International Consortium of FD/MAS invites you to its Film Screening and Community Discussion on the 24th of February at 7:30 pm EST. The event is free and is in…
A group of autoimmune diseases called anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV) is the subject of a preprint under consideration at Pediatric Rheumatology. ANCA-AAV is characterized by damage to small…
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