Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA…
Contact Tracing Contract Tracers are an essential component to stopping the spread of COVID-19. Essentially, it entails finding those exposed to the virus and quarantining them until they are no…
A recent report has documented a positive case study of a potential treatment option for one of the complications Marfan syndrome can cause. This report was published in the Journal of Oral Implantology. …
As reported in PressReleasePoint, Researchers at the University of Sheffield have found a clue to the cause of a set of rare neurological disorders known as leukodystrophies- an issue with…
Idiopathic Pulmonary Fibrosis Progression Veracyte has just signed a deal with Yale University which will help them develop the very first test which can predict disease progression for idiopathic pulmonary…
For those in the rare disease community, you know that it can take a long time to reach a diagnosis for your condition: an average of 4.8 years! But upon diagnosis, many…
The last decades have seen significant progress when it comes to medical care for rare diseases. Tasks doctors once slaved over were transformed with better speed and efficiency by modern…
Edwin Chapman, who works at the University of Wisconsin-Madison, has been studying neurons for most of his career. He and his team recently made a discovery regarding SYT1-associated neurodevelopmental disorder.…
Many patients with rare diseases require some sort of treatment or therapy to manage their conditions. In the case of atypical hemolytic-uremic syndrome, or aHUS, this therapy is dialysis. Learn…
By Natalie Homan from In The Cloud Copy The COVID-19 pandemic has changed the lives of millions around the world in an incredibly short span of time. It’s hard to…
By Natalie Homan from In The Cloud Copy Two businesses that advocate for patients with rare and orphan diseases announced recently that they are teaming up to help patients get…
May is recognized as Behçet's Disease Awareness Month and May 20th is recognized as Behçet's Disease Awareness Day. The focus of these events is to help spread awareness about this…
When studying biomarkers of lupus nephritis, researchers at the University of Houston discovered something interesting: race-based differences. Biomarkers are some sort of measurable substance; in this case, urinary proteins. However,…
Enanta Pharmaceuticals has recently released the results from the second phase of its INTREPID trial, which studied EDP-305 as a treatment for primary biliary cholangitis (PBC). The results were positive,…
The understanding of rare diseases is often lacking, making it difficult for rare disease patients to get the diagnosis and treatment that they need. Dr. Tracy M. French aims to…
According to a recent press release published by the National Institutes of Health (NIH), a new survey spearheaded by the Rare Diseases Clinical Research Network (RDCRN) is seeking to understand…
As reported in WMS, Kathy Miska was beginning to have difficulty walking. This time, she knew what was happening. She'd seen the same thing happen to her sister, who went…
A 6-year-old girl presented with facial abnormalities, developmental delays, and issues with her renal system. Her family took her to their family physician. Then, when that uncovered no cause,…
Protalix BioTherapeutics has recently announced the results from the third phase of the BRIDGE trial, which evaluated pegunigalsidase alfa for the treatment of Fabry disease. As these results were positive,…
As the world grows more technologically advanced, so does the medical field. Doctor-patient communication can happen over the phone or email. Telemedicine abounds. More technology is being pointed towards remote…
According to Juvenile Arthritis News, treating juvenile idiopathic arthritis early helps reduce symptoms, nearly "inactivating" the condition. The Canadian Alliance of Pediatric Rheumatology Investigators (CAPRI) JIA Registry found that early…
A recent study published in Blood Cancer Journal has established a new method for effectively predicting survival outcomes for patients with low-risk myelodysplastic syndromes (MDS). The method is known as the…
Running Wild for a Cure 5K Run-Walk Sunday, May 17th, 2020 This goal of this race is to raise funds for the Superficial Siderosis Research Alliance, a nonprofit organization dedicated…
May 16th is celebrated annually as Hereditary Angioedema (HAE) Awareness Day. The event was established with the goal of spreading awareness about this rare condition among both the general public…
May 16th is recognized as World Congenital Disorders of Glycosylation (CDG) Day. First launched in 2016 by the Portuguese Association for CDG with support from a variety of CDG patient…
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