According to a story from WXXI News, a startup from Rochester has set out to solve a dire medical problem that has global impacts on health. The problem at hand…
The United States Food and Drug Administration has awarded Rare Paediatric Disease Designation to an experimental drug for the treatment of Stargardt disease. For more information, you can find the…
According to a story from BBC, there are only around ten people in the world who are currently living with a replacement bladder that was derived from their own cells.…
According to a story from The New Paper, the KK Women's and Children's Hospital (KKH) in Singapore has successfully developed a test that could make diagnosing genetic rare diseases more…
Bone Therapeutics, who are developing an allogeneic bone cell therapy as a treatment for delayed-union fractures of long bones, has announced the results of a Phase 1/11A study. They have…
Three large black and white beasts rest under a shade tree to avoid the sun’s hot rays. They appear to be chewing gum, although they are really chewing their cud.…
According to a story from BBC, John Mason, age 80, is one of over 2,000 people in Wales who currently have idiopathic pulmonary fibrosis (IPF), a rare and ultimately lethal…
According to a story from Hematology Times, a recent study found that genetic sequencing after the completion of a hematopoietic stem cell transplant could provide doctors with vital information about…
Susan Aumiller’s story begins with a dream. She and her husband, Bob, made it a goal to visit every national park in the United States. About halfway through the journey,…
According to a story from Medical Xpress, three medical students at Florida International University are hoping to see if DNA collected from tampons could help detect ovarian cancer. There is…
Novartis has carried out a survey to find out what elements of a treatment are most important to patients with psoriasis. The survey found that patients value clear skin the…
According to a report by CheckOrphan, a recent study investigated the use of genome editing directly in the body. The research involved the use of these editing techniques in cases…
The United States FDA has awarded Breakthrough Therapy Designation to a drug called LOXO-292 for the treatment of RET-altered non-small cell lung cancer and thyroid cancer, in patients who meet…
The United States Food and Drug Administration has granted Breakthrough Device Designation to a medical device that is designed to screen for hyperkalaemia (high blood potassium levels) without taking a…
According to a story from Bangor Daily News, Andrea Dole decided to have a home birth for James, her first child. With the support of her partner, Patrick, two midwives,…
The United States Food and Drug Administration has awarded Fast Track Designation to the investigational drug miransertib (ARQ 092), which is being researched as a potential treatment for PIK3CA-related overgrowth…
The process of getting diagnosed with a rare disease, as well as living with one, can be a traumatic experience for patients. Many rare disorders or conditions may commonly cause…
According to a story from pm360online.com, the biopharmaceutical company Insmed Incorporated recently announced that the data from its Phase 3 clinical trial of amikacin liposome inhalation suspension (ALIS). ALIS was…
The first US patient has been dosed with the drug MIN-102 as part of a clinical trial into its effects in patients with adrenomyeloneuropathy. For more information about this, you…
Top-line data from a Phase 3 study of an experimental method of visualising prostate cancer has been announced. While it met the primary endpoint for specificity, it did not meet…
According to a report by The Telegraph, multiple sclerosis patients are being refused an important drug. The drug, known as ocrelizumab will not be funded by The National Institute for…
Recent medical developments have led to new drugs designed to treat rare genetic disorders. However, the high cost of many of these drugs limits the number of patients who have…
The pop-punk singer Avril Lavigne has announced her return to music with a new album that she made while fighting chronic Lyme disease. In a letter to her fans, she…
Azathioprine is a drug used in the treatment of a variety of conditions. Inflammatory bowel disease, arthritis, and vasculitis may all include azathioprine as part of their respective therapies. It…
A recent study published in the journal Cell has investigated the process behind the genetic condition Glut1 deficiency syndrome. Many genetic disorders are thought to be caused by genetic alterations that…
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