Adult Growth Hormone Deficiency (AGHD) is a rare condition when your body produces less growth hormone (GH) than normal for your age and sex. AGHD may affect your body systems,…
According to Biotech and Pharma Industry News, British pharmaceutical manufacturer Mallinckrodt has reported inconclusive results in its Phase 2/3 trial of VTS-270, a drug candidate for the treatment of the…
REGENXBIO is a biotechnology company that has been developing a technology platform for gene therapy called NAV. AAV9 is one of the vectors being tested with NAV technology as a…
Short bowel syndrome is a malabsorption disorder that causes the body to not get the fluids or nutrients it needs. It can cause dehydration and malnutrition, in addition to other…
Neuroendocrine tumors are growths which form from neuroendocrine cells in various places throughout the body. They can occur in the gastrointestinal track, the pancreas, the lungs, the thymus, and other…
Biobanks store biological samples to help scientists research certain diseases. Registries collect information from patients and standardize it, but no samples are stored. Which of these is best for rare…
Batten disease is a lysosomal storage disorder. It's neurodegenerative, meaning it results in the deterioration of neurons. One of the most common forms of this disease is caused by mutations in the…
According to a story from University at Buffalo, a recent discovery of a new genetic mutation continues to build on decades of research from Dr. Richard Gronostajski, a professor and biochemist…
According to a story from the Australian Jewish News, a Jewish teen named Gidon Goodman was born with Gaucher disease. Recently, he started the first Gaucher disease patient support group…
According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the…
According to a story from tradeshownews.vporoom.com, the biopharmaceutical company Viking Therapeutics, Inc., has recently released the results from a Phase 2 clinical trial. This trial is testing the company's investigational therapeutic…
According to a story from Benzinga, the drug developers AstraZeneca and Merck have recently announced that the US Food and Drug Administration (FDA) has decided to accept a supplemental New…
According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…
According to a story from UCI News, University of California Irvine scientists have developed an innovative immunotherapy that could screen patients in order to analyze the composition of their tumors…
According to a story from BioSpace, the gene therapy company Rocket Pharma recently announced that its investigational product candidate RP-L102 recently saw its Investigational New Drug (IND) application gain approval…
A $1.4 million dollar donation to the University of Massachusetts Medical School will help to fund research into a gene therapy for Tay-Sachs disease as it transitions from pre-clinical to…
The annual Global Genes Rare Patient Advocacy Summit is wonderful place for families affected by rare disease to come together and interact face to face with advocacy leaders in addition…
Some experiences are seen through rose-colored glasses while others are hazy or ambiguous. Many, we try desperately to remember while others we we wish we could forget. And a special…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc., recently announced the presentation of data which revealed that the company's proprietary virus vectors, which are…
I hope that you know a wonderful doctor. The type who listens wholly, respects you, validates your concerns and opinions, and goes above and beyond to make sure you get…
According to a story from EurekAlert!, researchers have been scrambling to find a useful treatment for acute flaccid myelitis, a rare disease that has been appearing in unusual numbers in…
According to a story from Scleroderma News, a recent study found that men were more likely to have severe scleroderma early in their diagnosis in comparison to women. This is…
Often times it seems that research for the rarest of conditions is brushed aside. Primary attention is given to more common illnesses which affect more people. For instance, in the…
A recent study has looked into the use of video visits with patients by clinicians in Sweden, to try to find out how doctors decide which patients to offer them…
As we age, we become more at risk for certain conditions. For instance, osteoporosis, atrial fibrillation, certain cancers, and heart disease, are more common in older individuals. These conditions pose…
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