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…
October is Gaucher Disease Awareness Month! The National Gaucher Foundation (NGF) has some wonderful resources that can come in handy for the Gaucher patient community. Let's take a look below!…
According to a post from gaucherdisease.org, clinical trials are an essential component to the development and testing of new therapies for Gaucher disease and other rare diseases. For some rare…
Outcomes of a pilot study on Gaucher disease using a mobile phone app and wearable technology demonstrate a more accurate approach to disease assessment. Gaucher disease is a lysosomal storage…
According to a story from The Globe and Mail, Canada's Patented Medicine Prices Review Board announced last month that drug prices would be subject to new regulations. These new rules…
According to a story from Direct Relief, when Yusra was just a year old, she began to have mysterious bouts of fever. Her family had no explanation. By age three,…
According to a story from Gaucher Disease News, the German drug company Centogene has recently released a free smartphone app called MyLSD which will allow patients with Gaucher disease monitor…
According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical need of its rare disease…
According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of…
This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…
Gaucher disease (GD) is a rare lysosomal storage disorder. It is caused by a deficiency in the glucocerebrosidase enzyme. In June, a new study was published in the International Journal of…
Rare Disease Fund Many families affected by rare diseases cannot afford the necessary treatments. Due to the small population of people these diseases affect, exorbitant price tags are often placed…
According to a story from the National Gaucher Foundation, the stress and difficulties of living with a rare or chronic disease can take a major toll on a patient's mental…
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In about five years India’s population will exceed that of China which is currently ranked number one according to population. Contrast these numbers with the fact that only ten percent…
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