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Zolgensma is a gene therapy developed by AveXis for the treatment of spinal muscular atrophy. It was approved by the FDA in May of 2019, and was later cleared for…
Phase IIa of the clinical trial of Lu AG06466 has recently concluded. This drug is meant for the treatment of Tourette syndrome, but has not met the primary endpoints in…
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…
In June 2019, Dicerna Pharmaceuticals submitted a clinical trial application for their DCR-A1AT therapy and expects to treat the first patient with A1AD-related liver disease near the end of…
As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…
The world is experiencing an unprecedented time in the medical realm with the rapid spread of COVID-19, a novel coronavirus. It may be difficult to understand how coronavirus may impact…
In an interview with Medgadget, Dr. Christy Sheehy and Dr. Zachary Helft of C. Light Technologies discuss up-and-coming technologies in the diagnosis and treatment of neurological diseases such as…
Mother of two, Mina Manchester, has been navigating raising two children with an immunocompromising disorder for four years. It's this unique experience that makes her feel more prepared and…
For generations, economists, pundits, and holders of public office have told the poor that pulling themselves out of poverty was just a matter of acquiring the right skills, making the…
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…
APR Applied Pharma Research is a small pharmaceutical company in Switzerland concerned with innovations for rare diseases. They have recently been granted the Orphan Drug designation by the FDA, meaning…
According to a story from Financial Buzz, the biotechnology company Inflazome recently announced that it has completed a phase 1 clinical trial. This trial was testing the company's experimental treatment…
Relapsing polychondritis is a rare disease which causes damage to the cartilage, trachea, and lungs. It's an autoimmune condition meaning an overzealous immune response causes the body to attack its own…
A recent study, conducted my Melinda Kavanaugh from the University of Wisconsin, Milwaukee, examined the role of children who are acting as caregivers for those diagnosed with amyotrophic lateral sclerosis…
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…
Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…
In May of 2019, the FDA approved a new therapy for Spinal Muscular Atrophy (SMA) called Zolgensma. Now, that therapy has been approved by the Japanese Ministry of Health, Labour…
Published in Social Science & Medicine, a recent study conducted by Drexel University has shed light on how, even in a world of evolving precision medicine, there is also merit in…
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…
Is it safe for those with autoimmune rheumatic diseases to undergo the termination of a pregnancy? According to research published in Arthritis & Rheumatology, the answer is yes. Learn more…
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…
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