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…
According to a story from stuff.co.nz, a two year old boy named Rhys from Auckland, NZ has spent much of his young life in the hospital. This is because he…
According to a story from BioPorfolio, a recent study first published in STEM CELLS Translational Medicine describes a unique stem cell procedure that has the potential to be a major step…
Current treatment for Huntington's disease only relieves symptoms, allowing for the progression of the disease to continue. UniQure, a company that works with gene therapies, aims to change this. They…
As originally reported in Yahoo Finance, the coronavirus is disrupting many of our expectations of how we planned to live out the next few months. Some frequenters of the hospitals…
According to a story from Medpage Today, rheumatologists could play an important role in treating patients with the most severe cases of COVID-19/coronavirus. This is because some of the drugs…
Molly Ouimet was three years old when she got her first kidney stone. Molly, who wasn't very vocal at the time, had a difficult time explaining the pain that she…
According to a story from mdmag.com, a phase 2 trial testing the drug avapritinib as a treatment for systemic mastocytosis is currently ongoing. The results so far suggest that the…
According to a story from Benzinga, the Novartis Company AveXis recently announced the results of long term studies testing the impact of Zolgensma, a gene therapy treatment that was approved…
According to a story from The Intercept, Gilead Sciences' antiviral drug remdesivir, which is being tested as a treatment for coronavirus/COVID-19, was just given Orphan Drug designation by the US…
As originally reported in European Pharmaceutical Manufacturer Magazine, Chiesi, an Italian pharmaceutical company, has announced they will be launching a new division for rare disease research and therapy innovation. The…
If the world were as careful in everyday life Perhaps I’d be sick less, I’d feel much less strife It's the small precautions, like washing your hands That for me…
Biological engineers at MIT are using a new technique to study various organs and their interaction with our immune system. According to a recent article in Science Daily, the researchers…
Identifying biomarkers may be an improved method of classifying Alzheimer’s and Parkinson’s. As outlined in a recent article covered by Parkinson's News Today, both diseases are diagnosed for the most…
By Jodee Redmond from In The Cloud Copy Massachusetts Institute of Technology (MIT) researchers have recently announced they have identified hundreds of genes that are required for neuron survival. The…
Sunday, March 15, 2020 was the sixth International Stiff Person Syndrome (SPS) Awareness Day. The patients who often are forgotten on this day of awareness are the rarest, most severely…
859 words vs 970 words 8% An article recently published in Science Daily quotes the results of a study showing a favorable outcome for six out of nine x-linked chronic…
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