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…
From Friday, June 7th to Sunday, June 9th, 2019, I had the privilege of attending the 2019 International Waldenstrom's Macroglobulinemia Foundation Educational Forum. This year, the annual event was held…
According to a story from Global Genes, the drug developer SpringWorks Therapeutics announced that the US Food and Drug Administration (FDA) has granted the company Fast Track Designation for its…
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…
Emily Ventura has never met in-person another person living with progressive familial intrahepatic cholestasis (PFIC), her daughter’s life-threatening ultra-rare genetic disease. That will change on June 21st when Emily and…
Happy Thursday! Today, we're highlighting stories on three patients facing different struggles: a professor, a Jeopardy host, and a man from New Jersey. We also have an article about a…
According to a story from globenewswire.com, the biopharmaceutical company Denali Therapeutics, Inc. has recently announced that its experimental product candidate DNL310, which is currently being developed as a treatment for…
According to a story from Alabama News Center, a scientist affiliated with Southern Research, Dr. Rita Cowell, has been hard at work researching new potential treatments for amyotrophic lateral sclerosis,…
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…
According to a story from Business Wire, the biopharmaceutical company X4 Pharmaceuticals, Inc. and the medical genetics company Invitae Corporation have recently announced that they have come into a parternship…
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…
According to a publication from Yahoo News, five doctors interviewed by the media organization have reported meeting with a number of migrants detained by United States Customs and Border Protection…
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.…
According to a story from news-medical.net, an international research team affiliated with the UK Medical Research Council, Indiana University School of Medicine, and the University of Kansas have released a…
According to a publication from Fierce Biotech, Biotech company Sangamo recently updated investors on three trials it has underway testing experimental forms of gene and cell therapies. The trials are…
According to a press release from Alnylam Pharmaceuticals, the Massachusetts-based biopharmaceutical company has completed the submission of its New Drug Application (NDA) for givosiran — its experimental acute hepatic porphyria…
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…
Do you have ADPKD? If you or someone you know has ADPKD, email us at [email protected]. We'd love to hear from you about your experience! According to a story from…
"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)…
According to a story from Bloomberg, the company Cadent Therapeutics recently announced that the company's experimental product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…
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…
According to a story from Science Nordic, the monoclonal antibody drug rituximab (often marketed at Rituxan) had no effect on patients with chronic fatigue syndrome (CFS). Chronic fatigue syndrome is…
Happy Thursday! Today, we're highlighting four articles on rare disease news. First, we have a story on a lawsuit against controversial stem cell treatments. Next, we have an article on…
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