According to a story from abc.net.au, parents Kellee and Jamie Clarkson of Queensland, Australia are calling for the implementation of newborn screening in the state for the rare disorder spinal…
In a story for Hemophilia News Today, author Shellye Horowitz has written a piece titled "An Open Letter to Providers Treating Women with Hemophilia." Women still face discrimination and unequal…
According to a story from abc30.com, Tawny Drain, now 33 years old, was in the sixth grade when she first started having symptoms of a rare illness called Caroli disease.…
According to a story from Nature, Dr. Carlos Heras-Palou was diagnosed with hATTR amyloidosis when he was just 39 years old. Initially, the devastating diagnosis was completely overwhelming, as the disease…
According to a story from the Children's Organ Transplant Association, the Fitzmaurice family of St. Louis, Missouri will have a ton to be thankful for as the holidays roll around…
According to a story from Daily Nation, Beneddettah Wangui of Kenya had been experiencing chronic pain since her teen years when she was finally diagnosed with fibromyalgia in 2012. However,…
According to a story from Medical Xpress, a team of researchers affiliated with the University of Granada and the research organization CIBERFES have discovered a pair of new mutations linked…
According to a story from governmenteuropa.eu, Professor Susanne Kircher, who is associated with the Austrian MPS Society and the Medical University of Vienna, writes about the state of rare diseases…
According to a story from stltoday.com, the Saint Louis Walk for PKD (polycystic kidney disease) took place on Sunday, October 13th, 2019. This event was just one of many that…
According to a story from messenger-inquirer.com, ten year old Abrielle Haynes of Sacramento, CA is often described as a 'china doll' by both family and friends. This is because she…
According to a story from pharmanewsdaily.com, the gene therapy company Avrobio has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to AVR-RD-02, an…
According to a story from scienceblog.com, a recent study has revealed that large portion of cystic fibrosis cases recorded in the Dominican Republic and Puerto Rico are linked to genetic…
According to a story from tmcnet.com, Alkermes plc and Biogen, Inc. have recently announced that the US Food and Drug Administration (FDA) has approved a new treatment for multiple sclerosis.…
According to a story from NBC News, the US Food and Drug Administration (FDA) issued an announcement during the summer detailing a patient death linked to the use of fecal…
According to a story from cancernetwork.com, the experimental drug selumetinib, which is currently licensed to AstraZeneca, has showed positive results in the phase 2 clinical trial. In this study, the…
According to a story from statnews.com, the approval of the new cystic fibrosis drug Trikafta has caused quite a buzz in this rare disease community. While this new drug offers…
According to a story from us.acrofan.com, the biopharmaceutical company AstraZeneca recently announced the release of positive results for a clinical trial testing the company's drug durvalumab (marketed as IMFINZI) in…
According to a story from Medical Xpress, an international team of scientist from 34 different countries are putting their heads together to identify the most effective treatments for ulcerative colitis,…
According to a story from shrewsbury.wickedlocal.com, biomarker testing has now become an important step for the treatment of cancer. In the past, most cancer treatments were based on where the…
According to a story from The Washington Post, data regarding the recently approved drug Trikafta, which is designed to treat cystic fibrosis, was so convincing that the US Food and…
According to a story from newswise.com, biologic medicines have had a major positive impact on outcomes for patients with the rare disease juvenile idiopathic arthritis (JIA). However, as these medicines…
According to a story from milanmirrorexchange.com, Lincoln Hopper, who will be turning two years old soon, was recently admitted to the hospital after showing indicators of heart failure. This is…
According to a story from Cape Town Etc, two girls in South Africa were living with the same rare disease: primary immunodeficiency (PI), a group of rare conditions in which…
According to a story from Fabry Disease News, a recent study has found that certain cells tend to age more rapidly in patients with Fabry disease when compared to unaffected…
According to a story from Buzzfeed News, Facebook has been receiving more negative press than ever lately, and so far it often seems justified. From the Cambridge Analytica scandal to…
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