According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals has recently announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its…
According to a story from BioSpace, the biopharmaceutical company MacroGenics, Inc. has just announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's…
In a press release from Eli Lilly and Company, the company has announced recently that the US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (BLA)…
According to a story from Multiple Sclerosis News Today, the drug company Bristol-Myers Squibb recently announced that its drug ozanimod (marketed as Zeposia) is now available for public use in…
According to a story from PR Newswire, the National Organization for Rare Disorders (NORD) has recently awarded a total of eleven new grant awards that will help fund essential research…
According to a story from EurekAlert!, a recent international study is utilizing data from 45 patients to describe a mostly unknown new rare genetic disorder. The researchers are recommending that…
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…
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