Researchers have recently uncovered two new rare diseases caused by the same mutation. This recent discovery is giving more families answers, and should hopefully begin to improve patient outcomes. The…
Wiskott-Aldrich Syndrome Wiskott-Aldrich Syndrome (WAS) is a rare immune disorder which causes platelets in the blood to function abnormally. It also causes autoimmunity. Patients suffer from severe bleeding which can…
As the medical field continues to advance, treatments are becoming less and less "traditional" because researchers are uncovering unique ways to combat illnesses. Therapies range from pills, to surgery, to…
Stargardt Disease Stargardt disease is a rare disease which results in progressive vision loss. It is also referred to as juvenile macular degeneration. Most often, the disease presents during childhood.…
Primary Sclerosing Cholangitis (PSC) and Autoimmune Hepatitis (AIH) are both rare and chronic liver diseases. They cause the bile ducts in the liver to become inflamed, resulting in severe liver damage.…
Hypereosinophilic Syndrome Hypereosinophilic syndrome (HES) is a rare disease which causes high levels of white blood cells in the body. These cells are called eosinophils, and they're essential for proper…
There was a time when most children diagnosed with a rare condition did not live until adulthood. But now, with advances in medicine, many individuals are living long, full lives…
Within the pharmaceutical industry, we need 1) better policies for patients and 2) improved communication/education of the policies that do exist for patients and the rights that they have. Ultimately,…
According to a story from BioSpace, the biotechnology company Viela Bio recently announced that the US Food and Drug Administration (FDA) has awarded an experimental therapy in development by the…
The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…
Happy Thursday! We hope everyone's spring is off to a good start! As the weather warms up, we're sharing stories demonstrating patient hope, strength, and resilience. We start with a…
Neurodegenerative Diseases There are a wide range of neurodegenerative diseases including Huntington's disease and Parkinson's disease. These conditions typically affect the older population. As the world's population ages, more people…
Background Novartis ended all of their gene therapy programs which were oncology related in 2016. Unfortunately, that meant an end to many programs that still had potential. This included cell…
According to a story from globenewswire.com, the gene therapy company Fibrocell Science, Inc. and the biopharmaceutical company Castle Creek Pharmaceuticals have recently announced a collaboration between the two companies. The…
According to a story from Top Class Actions, the drug companies Biogen, Inc. and Abbvie recently released a recall for their multiple sclerosis drug Zinbryta following several reported cases of…
Results from the largest United States study to date of Lambert-Eaton myasthenic syndrome (LEMS) have indicated that for the majority of patients, Firdapse is the most effective treatment option currently…
Researchers at GENFIT are excited about promising results from studies investigating a new potential therapy for PBC and NASH, two rare liver diseases. Primary Biliary Cholangitis Primary biliary cholangitis (PBC)…
According to a story from globenewswire.com, the biopharmaceutical company TG Therapeutics, Inc. recently made an announcement declaring that the US Food and Drug Administration (FDA) had awarded the company Orphan…
When you’re diagnosed with a rare condition it can change the entire course of your life, at least at the beginning. But those living with diseases like these are fighters.…
According to a publication from KYN24, the Food and Drug Administration recently approved mucopolysaccharidosis type VII (MPS VII) drug Mepsevii for marketing and administration to patients in the United States.…
Severe Combined Immunodeficiency Severe Combined Immunodeficiency (SCID) is a rare disease which causes patients to practically have no immune system at all. That means that for these patients, even a…
Alex Knoll Alex Knoll is not your average 14-year-old. In addition to being a student, he's also an inventor, entrepreneur, app creator, and public speaker. Most of the Idaho resident's…
Madison Ricci Madison Ricci is a 19-year-old who was diagnosed with Lupus at the age of 12. Lupus is a rare disease in which the body’s immune system begins attacking…
LLUCH Loma Linda University Children's Hospital (LLUCH) is one of six hospitals in the University's health system. They are a faith-based system located in Southern California. They are the only…
According to a story from globenewswire.com, Vertex Pharmaceuticals Inc. and CRISPR Therapeutics have recently announced that the US Food and Drug Administration (FDA) has awarded CTX001, an experimental gene-edited stem…
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