Rare Disease Fund Many families affected by rare diseases cannot afford the necessary treatments. Due to the small population of people these diseases affect, exorbitant price tags are often placed…
The FDA has approved a new therapy for chronic immune thrombocytopenia (ITP). This treatment is an oral therapy to be taken with food, created by Dova Pharmaceuticals. It is called…
Frankie Jr. Muretic Frankie Jr. Muretic lives in Mississauga in Ontario, Canada. One morning he woke up with a rash, causing his parents concern. They brought the five-year-old boy to…
Dicerna Pharmaceuticals has just announced that they have submitted a Clinical Trial Authorization application for their investigational therapy DCR-A1AT. This application has been submitted to the Swedish Medical Products Agency…
Inflammatory Bowel Diseases Inflammatory bowel diseases (IBD) is the overarching term for both ulcerative colitis and Crohn's disease. In Canada, there are around 270,000 individuals living with the one of…
Emily Dover This is Emily Dover's story. At birth- perfectly healthy newborn, smaller than her siblings (8 pounds) 1 week- was having trouble sleeping and began crying in pain 4…
Tannaz Motevalli Tannaz Motevalli is the Exhibition Coordinator at the National Library of Medicine, an artist, a researcher, and a writer. She also lives with cystic fibrosis (CF). Experiencing fewer…
Jane Biehl Jane Biehl has worked as a professor, rehabilitation counselor, and a librarian. She holds three different degrees, and spreads her knowledge through freelance writing. She also has a…
It is 2019, and minority populations still account for fewer than 10% of clinical trial participants. Two decades ago, the "NIH Policy and Guidelines on the Inclusion of Women and…
Eosinophilic Esophagitis (EoE) Eosinophilic esophagitis (EoE) is a rare and chronic disorder which affects the digestive system. Eosinophils are a type of white blood cell that help fight infections. This…
Chronic Kidney Disease Chronic kidney disease (CKD) is a rare condition which is defined as lasting and progressive kidney damage. The kidneys are responsible for filtering waste and fluids from…
IPain The International Pain Foundation (IPain) is a nonprofit organization dedicated to supporting those with chronic pain due to neurological, musculoskeletal, inflammatory, degenerative, and emotional conditions. They aim to improve…
At the 2019 American Thoracic Society Conference this year multiple presentations demonstrated the importance of the Pulmonary Fibrosis Foundation's Patient Registry. About the Registry This registry was created by the Pulmonary…
A recent article written by Craig Klugman debates the ethical obligations pharmaceutical companies have to conduct clinical trials for drugs which have a likely potential to benefit a patient population…
What are Generic Drugs? Generic drugs are any medications "equivalent" to brand name pharmaceuticals. These drugs are much cheaper than the brand name medications and the United States Food and…
The Importance of Braille For any child with a rare disease that leads to blindness or visual impairment such as Stargardt disease, Usher Syndrome, Alström syndrome, or Retinal Blindness life…
Blood Donations For many people living with a rare disease such as Beta thalassemia, Hemophilia, Aplastic Anemia, Myelodysplastic syndromes, Acute Promyelocytic leukemia, and others, blood transfusions are not uncommon. For…
Ella Casano is a 12-year-old living with Idiopathic Thrombocytopenic Purpura (ITP) who had a brilliant idea about how she could help other children living with rare diseases like her own.…
Stem Cell Therapies While stem cell therapies hold a great deal of promise for many rare diseases, there is still a lot that we don't understand. Experimental stem cell therapies…
Meredith Resnick's breast cancer risk is 42%. This risk is made up of her higher breast density (denser breasts are associated with increased risk), Ashkenazi Jewish heritage, a family history…
"A new way to improve the efficacy of a drug taken by millions of patients throughout the world." Background Adalimumab (also known as Humira) and infliximab (also known as Remicade)…
MVID Microvillus inclusion disease (MVID) is a rare, gastrointestinal disease which can cause impaired development in children. It is characterized by severe diarrhea which leads to dehydration and malnutrition. MVID…
Stargardt Disease Stargardt disease is a rare condition that causes vision loss due to the buildup of vitamin A byproducts in the retina. The byproduct causing the largest buildup is…
A recent study published in PLOS Biology has shown a promising new vulnerability within human viruses which could lead to the development of a new antiviral drug. This study was…
Transfusion-Dependent β-Thalassemia (TDT) Transfusion-dependent β-thalassemia (TDT) is a rare disease that is caused by a mutated β-globin gene. This mutation causes the amount of hemoglobin in the body to be…
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