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Updates on PR001, a Treatment for Gaucher Disease
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…
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New Therapy Being Studied for Treatment of Gaucher Disease
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…
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An Experimental Gene Therapy for Gaucher Disease has Earned Orphan Drug Designation
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…
Study: Stem Cell Therapy Shows Sign of Efficacy for Gaucher Disease Treatment
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!
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!…
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A Primer on Clinical Trials for Patients With Gaucher Disease and Other Rare Illnesses
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…
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Controlling Drug Prices Shouldn’t Harm Rare Disease Patient Access to Treatment
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…
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Tanzanian Girl With Gaucher Disease to Receive Treatment Through Critical Program
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,…
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Drug Company Releases App to Monitor Biomarkers for Gaucher Disease Patients
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…
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India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases
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…
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Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders
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…
Gaucher Disease Patient and Activist Successfully Improves the Care for Other Rare Disease Patients in Her Country
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…
Recent Study Indicates Lyso-Gb1 is an Extremely Effective Biomarker for Monitoring Children with Gaucher Disease
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…
Singapore Launches the Rare Disease Fund to Help Patients Cope with Exorbitant Drug Costs
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…
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Gaucher Disease and Mental Health: Facing the Challenges
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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Rare Diseases: Only 10% of India’s 1.37 Billion People Have Health Insurance
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…
Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa
The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…
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The Impacts of Gaucher Disease on the Skeletal System and the Future of Treatment
According to a story from the National Gaucher Foundation, a significant challenge for many patients with Gaucher disease are the effects that the illness can have on bones. Common treatments…
Gaucher Disease Discovery Could Benefit Parkinson’s Disease Patients
When one rare disease is found to be in some way similar to another rare disease, research can often be accelerated for both. For instance, recent discovery by a professor…
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Woman with Gaucher Disease Aims to be a Source of Encouragement for Other Patients
According to a story from Gaucher Disease News, life for 24 year old Madeline Collin, who has Gaucher disease, has never been easy. However, being subject to bullying and a…
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Critical Treatment for Gaucher Disease Gets Expanded Coverage in South Korea
According to a story from Korea Biomedical Review, the Sanofi Genzyme Korea, a subsidiary of the pharmaceutical company Sanofi, recently issued an announcement stating that the South Korean Ministry of…
China Continues to Amend Policies to Improve Care for Rare Disease Patients
Problems in China Around the world there are 7,000 rare diseases currently identified. Of these, researchers have uncovered effective treatment options for less than 10%. Sadly, even when diseases have…
The Argument for Mandatory Newborn Screening and Government Subsidies for Rare Diseases in India
The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…
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Study Suggests that Spleen Artery Aneurysms May be more Common in Gaucher Disease than Previously Thought
According to a story from Gaucher Disease News, a recently published case report study suggests that instances of splenic artery aneurysm are being underreported in patients with Gaucher disease. The…
New Hope for Rare Disease Patients
Rare disease isn't as rare as it sounds. Ten percent of Americans have a rare disease-- and there are almost 7,000 known rare diseases. A rare disease, also known as…
