New Trappsol Cyclo Safety and Efficacy Data Available for NPC1
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New Trappsol Cyclo Safety and Efficacy Data Available for NPC1

On July 30, 2021, biopharmaceutical company Cyclo Therapeutics shared that it had released new and promising data regarding the safety, efficacy, and tolerability of Trappsol® Cyclo™ ("Trappsol Cyclo") for patients with…

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This Drug Saves Niemann-Pick Disease Type C Patients. It’s Being Pulled in Six Months.
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This Drug Saves Niemann-Pick Disease Type C Patients. It’s Being Pulled in Six Months.

According to a story from Scary Mommy, mother Sara McGlockin recently received devastating news: the drug that was successfully treating her five year old daughter Marian was being discontinued in…

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Parents Receive News That Their Child’s Niemann-Pick Disease Medication Will be Discontinued

Marian McGlockin was eighteen months old when she was diagnosed with Niemann-Pick Type C disease, a rare and fatal disorder. Marian could barely speak and was unable to walk. According…

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New Drug Application for Niemann-Pick Disease Type C Treatment
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New Drug Application for Niemann-Pick Disease Type C Treatment

Orphazyme has recently submitted their New Drug Application to the FDA for their Niemann-Pick disease type C (NPC) treatment, arimoclomol. This application comes after the biopharmaceutical company was already granted…

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