According to a story from The Intercept, Gilead Sciences' drug remdesivir is being touted in the media as a major development in the treatment of COVID-19, the illness caused by…
According to a story from Sarcoidosis News, while the global challenge of the COVID-19 pandemic has been an obstacle for advancement in many rare diseases with trials and research grinding…
According to a story from Neurology Advisor, a recent study has found that patients living with neuromyelitis optica spectrum disorder (NMOSD) are at an increased risk of experiencing bone loss,…
According to a story from globenewswire.com, the biopharmaceutical company Apellis Pharmaceuticals, Inc. has announced that it is currently making plans to submit a New Drug Application (NDA) to the US…
According to a story from UNC Health Care, the Belgian drug company Argenx recently announced that its investigational treatment efgartigimod has performed well in a phase 3 clinical trial. The…
According to a story from globenewswire.com, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the…
According to a story from Parkinson's News Today, an analysis of results from several different clinical trials suggests that the drug istradefylline (marketed as Nourianz) can reduce the duration of…
According to a story from Parkinson's News Today, the Vercise system, developed by Boston Scientific as a method of deep brain stimulation, was able to provide benefit to Parkinson's disease…
According to a story from Medical Xpress, an Italian study has found that people in urban areas are at a 29 percent greater risk of developing multiple sclerosis (MS). This…
According to a story from Parkinson's News Today, ropinirole (marketed as Requip) skin patches proved capable of improving motor function in patients with advanced Parkinson's disease when compared to placebo.…
According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…
According to a story from Pulmonology Advisor, a recent study has evaluated risk factors for the appearance of bronchiectasis in patients with aspirin-exacerbated respiratory disease (AERD). A small number of…
According to a story from Guru Focus, the biopharmaceutical company Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. recently announced that the Ministry of Health of Spain has cleared…
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…
According to a story from Sanfilippo News, the healthcare tech company RDMD has recently announced that it has raised $14 million in Series A financing that will be dedicated towards…
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…
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…
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…
May 15th is recognized each year as Mucopolysaccharidosis (MPS) Awareness Day. The goal of this day is to help spread awareness about this group of diseases among the general public…
According to a story from abc7.com, a recent case study has identified what scientists believe to be the earliest case of a pediatric COVID-19 patient presenting with symptoms of pediatric…
The month of May is recognized as Myositis Awareness Month. The ultimate goal is to help spread awareness and educate both the general public and the healthcare field about myositis,…
May is recognized annually as Cystic Fibrosis (CF) Awareness Month. Each year, this rare disease community comes together in order to help spread awareness about cystic fibrosis among the general…
According to a story from Xconomy, the drug company PTC Therapeutics has come to an agreement that will allow it to acquire Censa Pharmaceuticals and its lead product candidate. This…
De acuerdo a una historia de PR Newswire, La Organización Nacional de Trastornos Raros (NORD, por sus siglas en inglés) acaba de lanzar un programa de ayuda crítica COVID-19. En…
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