New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review
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New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review

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…

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Arimoclomol Receives the Fast Track Designation for the Treatment of ALS
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Arimoclomol Receives the Fast Track Designation for the Treatment of ALS

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…

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Sanofi is Making Strides in Lysosomal Storage Disease Research

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.…

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An Experimental Gene Therapy for Gaucher Disease has Earned Orphan Drug Designation
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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…

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A Primer on Clinical Trials for Patients With Gaucher Disease and Other Rare Illnesses
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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
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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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India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases
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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
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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…

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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…

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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…

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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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