Compiling data, especially rare disease data, can be extremely helpful for research purposes and for doctors treating patients with that condition. Recently, the International Gaucher Alliance and Cerner Enviza have teamed…
One of the burdens that rare disease patients often face falls into the financial category, and that's treatments that come at a very high cost. One patient, a young girl…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
From November 21st until the 23rd, medical professionals and others involved in the metabolic field will come together for the 14th International Congress of Inborn Errors of Metabolism (ICIEM). It…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare or life-threatening illnesses, defined as those affecting under 200,000 people or…
In a recent news release, rare disease-focused biopharmaceutical company CANbridge Pharmaceuticals Inc. ("CANbridge") shared that the National Medical Products Administration (NMPA) of China accepted an Investigational New Drug (IND) application…
In mid-April, Kantar Health and the International Gaucher Alliance (IGA) shared the launch of a rare disease registry developed by the partnership. The registry, known as GARDIAN, will explore Gaucher…
At the American Society of Hematology Annual Meeting in 2020 (ASH 2020), the latest info and data related to Gaucher disease, a form of lysosomal storage disease, was presented. It…
Prevail Therapeutics, a company that focuses on developing gene therapies for neurodegenerative diseases, has announced that a composition of matter patent has been issued for PR001. This therapy is intended…
According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…
According to a story from Parkinson's News Today, a recent study may have discovered the origins of chronic pain and damage to the sensory nerves as found in some cases…
Gaucher Disease Awareness Month takes place every October, but amidst this new global pandemic, planning has taken place early to figure out how to make the month as successful as…
CytRx Corporation has released the results of the second phase of their study of arimoclomol as a treatment for Gaucher disease. The data was positive, and it demonstrated this therapy's…
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…
CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis…
Have you been feeling a little stiff or achy later? Maybe tired? I know that my body has undertaken a lot of stress thanks to the new life changes -…
Rare, neurologic diseases often do not have viable treatments. Gaucher disease and Parkinson's disease are two examples. In situations like these, medical professionals look to treatments for other conditions, such…
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.…
According to a story from the National Gaucher Foundation, the use of gene therapy in order to treat Gaucher disease has long been floated in medical theory, but only in…
Prevail Therapeutics has released an update on their Parkinson's disease treatment, PR001. They are currently dosing participants, and enrollment remains open. As of now, the study remains on track. Results…
Prevail Therapeutics has recently been granted the Orphan Drug designation by the FDA for their gene therapy PR001, which is intended to treat Gaucher disease. This treatment has also received…
Prevail Therapeutics is the creator of PR001, which is a gene therapy used for the treatment of Gaucher disease. They expected to begin dosing in the first half of…
Prevail Therapeutics recently submitted an Investigational New Drug application for their gene therapy called PR001. The FDA has recently notified them that this application is active, meaning that Prevail Therapeutics…
According to a story from pharmanewsdaily.com, the gene therapy company Avrobio has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to AVR-RD-02, an…
Stem cell therapy via intravenous injection lessened symptoms and prolonged survival of Gaucher disease, reports a study using a mouse model. The study was published in Human Molecular Genetics, and reported…
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