Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 3)
Courtesy of Debbie Kaflowitz

Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 3)

To learn more about Rachael's story, Niemann-Pick type C (NPC) symptoms, the diagnostic process, and how her mother Debbie found and offered support to other families, take a look at Parts…

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Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 2)
Courtesy of Debbie Kaflowitz

Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 2)

In Part 1 of Rachael's story, I spoke with her mother Debbie about what Niemann-Pick type C (NPC) is, the beginning of the diagnostic journey, and how Dr. Behar helped…

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Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 1)
Courtesy of Debbie Kaflowitz

Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 1)

“Hi, I’m Rachael, and I’m forever 33.” When Debbie Kaflowitz remembers her daughter, she thinks about everything that Rachael loved to do. Rachael enjoyed dancing and performing in ballet recitals,…

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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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