In this time of cancellations, closures, and shutdowns, many companies and individuals have found unique and creative ways to shift their services online. Better yet, some have temporarily waved their…
A recent study published in the Orphanet Journal of Rare Diseases examines the psychological burden of mitochondrial diseases in children that are suspected of having some form of it but have yet…
According to a story from Mirror Online, Vera Twomey from Cork, Ireland, was devastated when her four month old daughter had a severe seizure that lasted 45 minutes. Before long,…
According to a story from Hindawi, a recent study focused on congenital central hypoventilation syndrome serves to bring to light the importance of genetic testing of close family members based…
What is Repurposing? Repurposing involves testing whether or not a compound approved of another condition may be capable of also treating an illness with no approved treatment option. It's advantageous,…
Dr. Pierre Fenaux, of Hôpital Saint Louis, Paris and lead author of the MEDALIST trial, told Ash Clinical News that treatment with the investigational drug, luspatercept, improved the rate of…
According to a story from Sleep Review, the drug company Jazz Pharmaceuticals plc has recently filed a New Drug Application (NDA) for its investigational treatment JZP-258, which is being developed…
Emily Knakmuhs's two-and-a-half-year-old Kennedie had just made it past a tough couple of years. When she was born, her family noticed she wasn't growing, but her pediatrician attributed it to…
As originally reported in News Medical, a new study suggests that gastric bypass bariatric surgery may be the most effective treatment for non-alcoholic fatty liver disease (NAFLD). This disorder currently…
Hi everyone, We hope you're having a peaceful quarantine and taking care of yourself. As we all know, updates about COVID-19 is dominating the news right now. Of course, infomation…
According to a story from pharmaphorum.com, Jim Kuhn had a successful career in business for over 30 years, but everything changed a decade ago when he was first diagnosed with…
As originally reported in Fabry Disease News, the potentially fatal genetic disorder Fabry disease can be diagnosed most effectively in infancy by newborn screening programs. By identifying the disease early,…
In a Special Issue of the journal Disease Models and Mechanisms Highlights, James J. Dowling et al describes pediatric neuromuscular diseases as mostly genetic and affecting areas of the peripheral…
Pam Kelley, a reporter whose focus is on the challenges facing people below the poverty level, submitted this story to the Charlotte Magazine. Pam conducted interviews with Cheryl Potts over…
When Kyle and Sue Dempsey's daughter Mackenzie was three years old, she was in a car accident that resulted in head trauma. A couple of years later, when she was…
According to a story from People's Pharmacy, US Surgeon General Jerome Adams recently stated in a tweet that regular citizens should stop buying face masks because they are not useful…
A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…
According to a recent article published in Pharmaceutical-Technology, uniQure’s gene therapy AMT-130 may lower levels of the huntingtin protein that causes the disorder. An abnormal gene is responsible for damage…
A recent Biospace article covers a news release from 4D Pharma in Leeds, England. 4D has announced the first worldwide clinical confirmation of a biotherapeutic product. MRx0518 has been combined…
TARGET PharmaSolutions Inc. recently announced enrollment of the first patients in its ten year, ten thousand patient asthma study. Professor Michael Wechsler, M.D. pulmonologist at the National Jewish Health organization…
As originally reported in Porphyria News, rare diseases have historically been neglected by traditional medicine; their obscure and often genetic nature made them lack priority, stuck behind a long list…
According to a story from Buzzfeed News, Sharon Greenstein, who lives with the rare autoimmune disease known as Sjögren's syndrome, can no longer find the drug that she uses for…
Hi everyone, We know you might be overwhelemed with virus news-- and next week, we'll switch the theme up. For now, we wanted to send three updates your way about…
Researchers from the Murdoch Children’s Research Institute (MCRI) have recently discovered a previously unknown and uncharted neurodegenerative disorder. This rare disorder, which has not yet been named, affects children. It…
Patient Worthy's main focus is to share information and help patients in any way we can. We recently saw this video and thought that it was relevant for our followers. Created by Snow Companies, it is a virtually co-created user-generated video…
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