Study: Arimoclomol Shows Positive Results in the Treatment of Niemann-Pick Disease
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Study: Arimoclomol Shows Positive Results in the Treatment of Niemann-Pick Disease

  Orphazyme, a biopharmaceutical company, has recently released the data from their study of arimoclomol. This product is meant for the treatment of Niemann-Pick disease type C (NPC). Orphazyme specializes…

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These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies
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These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies

According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead…

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Found: Pelizaeus-Merzbacher Disease, the Cause, and a Potential Cure

  According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…

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Orphazyme Preps to File European Marketing Application for Experimental Niemann-Pick disease Type C Drug
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Orphazyme Preps to File European Marketing Application for Experimental Niemann-Pick disease Type C Drug

According to a press release from Danish biopharmaceutical company Orphazyme, the Company intents to proceed with a marketing authorisation application (MAA) for its experimental Niemann-Pick disease type C treatment arimoclomol.…

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Gene Therapy for 12 Rare Diseases will be Investigated Thanks to this Industry-Academia Partnership

Extended Collaboration  Amicus Therapeutics has just announced that they are expanding their collaboration with the University of Pennsylvania's Perelman School of Medicine for the next five years. This collaboration is…

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The Struggles of being a Rare Disease Patient in China

The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…

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Observational Study Conducts Long Term Follow up of Niemann-Pick Disease Type B Patients
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Observational Study Conducts Long Term Follow up of Niemann-Pick Disease Type B Patients

A study published in the Orphanet Journal of Rare Diseases monitored the long-term progression of 16 patients that had Niemann-Pick Disease type B. The study served to gather important observational data…

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Study Hopes to Improve Diagnosis of Niemann-Pick Disease and Other Rare Conditions

The first step in treating rare diseases is accurately diagnosing them. Unfortunately far too many people go years without knowing what is going wrong in their body. Doctors are trained…

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Possible Treatment for Niemann-Pick Disease Type C Performs Well in Clinical Trial

According to a story from labiotech.eu, the biotechnology company Orphazyme has been developing an experimental therapy for Niemann-Pick disease type C. In a recent Phase 2/3 clinical trial, the investigational…

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Results from Phase II/III Trial Bring Hope for FDA Approval for New Niemann-Pick Disease Type C Treatment

Niemann-Pick Disease is a genetic lysosomal storage disease. Lipids accumulate in the lysosomes of cells, causing debilitating symptoms. There are three forms of the disease- A, B, and C. Each…

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Potential Treatment for Niemann-Pick Disease Type C Granted Orphan Drug Designation
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Potential Treatment for Niemann-Pick Disease Type C Granted Orphan Drug Designation

Niemann-Pick Disease Type C is a lysosomal storage disorder caused by malfunction of the NPC1 protein. This protein's dysfunction results in a buildup of lipids in the lysosomes. As a…

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Potential Treatment for Niemann-Pick Disease is Granted Orphan Drug Designation in the EU
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Potential Treatment for Niemann-Pick Disease is Granted Orphan Drug Designation in the EU

According to a story from rdmag.com, the biopharmaceutical company IntraBio Inc., recently announced that its IB1000 series of investigational therapeutic compounds have been granted Orphan Medicinal Drug Designation as a…

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This Company Just Endorsed New Patient Advocacy Guidelines
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This Company Just Endorsed New Patient Advocacy Guidelines

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme has officially endorsed a set of guidelines that outline how rare disease patient advocacy organizations and drug companies should interact…

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Clinical Trial Results Look Promising For Experimental Niemann-Pick Disease Type C Treatment

According to a story from BioSpace, the biopharmaceutical company Orphazyme recently announced the results from its Phase 2/3 clinical trial of its investigational drug arimoclomol. This therapy is in development…

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Boy With Niemann-Pick Disease Type C to be Featured in Televised Charity Event
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Boy With Niemann-Pick Disease Type C to be Featured in Televised Charity Event

According to a story from the Milton Keynes Citizen, a local boy named Josh from the Milton Keynes community with the rare lysosomal storage disease Niemann-Pick disease type C will…

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Phase II Trial for Investigational Gaucher Disease Drug Begins
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Phase II Trial for Investigational Gaucher Disease Drug Begins

According to a story from GlobeNewsWire, the biopharmaceutical company Orphazyme A/S, which is committed to the development of treatments for patients that are living with rare diseases, has recently announced…

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Enrollment Completed in Niemann-Pick Disease Study!
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Enrollment Completed in Niemann-Pick Disease Study!

The Danish biotechnology company, Orphazyme, announced in early May of this year that enrollment was completed for its lead study program for Niemann-Pick type C disease. Niemann-Pick disease is a…

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