NPC – Patient Worthy

Rare Community Profiles: A New Treatment Option on the Horizon: Zevra Therapeutics’ Dan Gallo Discusses Arimoclomol, the NDA PDUFA Date, and the Needs of the NPC Community

Post author:Jessica Lynn
Post published:June 25, 2024
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Fotocitizen / Pixabay

Rare Community Profiles: The Urgent Need for a Cure for Niemann-Pick Type C: Two Parents Share Their Perspectives

Post author:Jessica Lynn
Post published:April 10, 2024
Post category:Niemann-Pick Type C Disease

Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Pexels / Pixabay

Phase 2 Study Evaluates Nizubaglustat for Niemann-Pick Disease type C

Post author:Jessica Lynn
Post published:March 29, 2024
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

There are limited therapeutic interventions for those living with Niemann-Pick disease type C (NPC), a rare and progressive genetic disorder. Unfortunately, many people with NPC experience severe and life-threatening complications…

source: pixabay.com

Study of the Week: Niemann-Pick Disease Type C Biomarker Could Help Diagnose Congenital Disorders of Glycosylation

Post author:James Moore
Post published:April 24, 2023
Post category:Congenital disorders of glycosylation Niemann-Pick Disease Niemann-Pick Type C Disease

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

source: pixabay.com

A Biomarker for Niemann-Pick Disease Type C Could Improve Congenital Disorders of Glycosylation Diagnosis

Post author:Jessica Lynn
Post published:April 17, 2023
Post category:Congenital disorders of glycosylation Niemann-Pick Disease Niemann-Pick Type C Disease

Let’s start with a little vocabulary lesson. Today’s word? Biomarker. A biological marker, or biomarker, refers to some sort of measurable indicator of disease. For example, blood pressure, cellular gene…

Patients with Nasopharyngeal Carcinoma May Benefit From the Chinese Herbal Medicine Shengmai Yin

Post author:Rose Duesterwald
Post published:March 9, 2022
Post category:Nasopharyngeal carcinoma

Nasopharyngeal carcinoma consists of cancers originating in the throat and the nose. Due to the way the cancers are positioned, radiation therapy may not be an option. Also, a number…

Don’t Dismiss Your Blocked Nose So Quickly: It Could Be NPC

Post author:Alyssa Stevens
Post published:February 22, 2022
Post category:Nasopharyngeal carcinoma

An article recently came out encouraging people who are experiencing a blocked nose to not just dismiss it as a common cold, as it could be nasopharyngeal cancer (NPC). Nasopharyngeal…

“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 3)

Post author:Jessica Lynn
Post published:December 2, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

In the first part of the "Save Our Medicine" series, you met moms Tiffany, DeAnna, and Sara, who are working to make a change and save experimental medication access for…

“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 2)

Post author:Jessica Lynn
Post published:December 1, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

For Part 2 of "Save Our Medicine," we continue talking with Tiffany, DeAnna, and Sara about their families' NPC journeys, as well as the push for medication access. If you…

Courtesy of Debbie Kaflowitz

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

Post author:Jessica Lynn
Post published:December 1, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

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…

“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 1)

Post author:Jessica Lynn
Post published:November 30, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

If your child was sick and there was a real, viable treatment option, the next step seems pretty straightforward: pursuing that option. But what if it wasn’t that easy? What…

Courtesy of Debbie Kaflowitz

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

Post author:Jessica Lynn
Post published:November 30, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

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…

Courtesy of Debbie Kaflowitz

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

Post author:Jessica Lynn
Post published:November 29, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

“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,…

Pexels / Pixabay

Study: Prevalence of Niemann-Pick Disease Type C

Post author:Kendall Mason
Post published:November 10, 2021
Post category:Niemann-Pick Type C Disease

It is often difficult to estimate the prevalence of a rare disease within the United States. For one, it can be difficult to find all of the patients with a…

Source: Elsa Burgos

INTERVIEW: Changing the Future for NPC

Post author:Jessica Lynn
Post published:September 29, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

In 1992, Elsa Burgos and her husband, living in Puerto Rico at the time, welcomed a new addition to their lives: their daughter Nicole. The pair were thrilled to have…

source: pixabay.com

Enrollment Begins for Phase 3 Niemann-Pick Disease Type C1 Trial

Post author:James Moore
Post published:July 16, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

According to a story from MarketWatch, the biotechnology company Cyclo Therapeutics announced that is has proceeded with the enrollment for its phase 3 clinical trial. This trial will evaluate an…

OLE Trial Data Shows Arimoclomol Efficacy for NPC

Post author:Jessica Lynn
Post published:July 7, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

In the past, arimoclomol has not always shown treatment promise. For example, the drug failed to meet primary or secondary endpoints in a clinical trial evaluating arimoclomol for patients with…

https://pixabay.com/photos/swing-playground-children-playing-1188132/

Audrey’s Story: Life With Niemann-Pick Disease Type C

Post author:Kendall Mason
Post published:April 12, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

Three people in the state of Indiana live with Niemann-Pick disease type C (NPC), and Audrey Mischler is one of them according to WTHI-TV 10. She was diagnosed very recently,…

source: pixabay.com

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

Post author:James Moore
Post published:April 9, 2021
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

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…

Trial of Niemann-Pick Disease Treatment is Expanded

Post author:Kendall Mason
Post published:November 18, 2020
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease Rare Disease

IntraBio has recently launched an extension study of their Phase II trial of IB1001, a treatment for Niemann-Pick disease Type C. This decision was made shortly after analyzing the positive…

source: pixabay.com

FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug

Post author:James Moore
Post published:September 18, 2020
Post category:Amyotrophic Lateral Sclerosis Gaucher Disease Inclusion Body Myositis Niemann-Pick Type C Disease

According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…

source: pixabay.com

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

Post author:James Moore
Post published:June 5, 2020
Post category:Amyotrophic Lateral Sclerosis Gaucher Disease Inclusion Body Myositis Niemann-Pick Disease Niemann-Pick Type C Disease

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…

Phase 1 Trial for Niemann-Pick Disease Type C Has Completed Enrollment

Post author:Trudy Horsting
Post published:October 25, 2019
Post category:Niemann-Pick Type C Disease Rare Disease

Niemann-Pick disease type C (NPC) is a rare disease caused by a defected NPC protein. In a healthy individual, this protein helps the body process cholesterol. Those diagnosed with NPC experience…

Pexels / Pixabay

This Company Just Provided a Grant for a Niemann-Pick Disease Type C Screening Program

Post author:James Moore
Post published:October 23, 2019
Post category:Niemann-Pick Type C Disease Rare Disease

According to a story from ir.stockpr.com, the biotechnology company Cyclo Therapeutics, Inc. (previously known as CTD Holdings) has recently thrown its support behind a Niemann-Pick Disease Type C newborn screening…

rawpixel / Pixabay

ICYMI: FDA Approves Expanded Access Application of Experimental Niemann-Pick Type C Drug

Post author:Scott Carlson
Post published:September 23, 2019
Post category:Niemann-Pick Type C Disease

According to a press release from CTD Holdings, the American Food and Drug Administration (FDA) recently approved an Expanded Access application from a physician to treat a pediatric Niemann-Pick disease…

Rare Community Profiles: A New Treatment Option on the Horizon: Zevra Therapeutics’ Dan Gallo Discusses Arimoclomol, the NDA PDUFA Date, and the Needs of the NPC Community

Rare Community Profiles: The Urgent Need for a Cure for Niemann-Pick Type C: Two Parents Share Their Perspectives

Phase 2 Study Evaluates Nizubaglustat for Niemann-Pick Disease type C

Study of the Week: Niemann-Pick Disease Type C Biomarker Could Help Diagnose Congenital Disorders of Glycosylation

A Biomarker for Niemann-Pick Disease Type C Could Improve Congenital Disorders of Glycosylation Diagnosis

Patients with Nasopharyngeal Carcinoma May Benefit From the Chinese Herbal Medicine Shengmai Yin

Don’t Dismiss Your Blocked Nose So Quickly: It Could Be NPC

“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 3)

“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 2)

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

“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 1)

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

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

Study: Prevalence of Niemann-Pick Disease Type C

INTERVIEW: Changing the Future for NPC

Enrollment Begins for Phase 3 Niemann-Pick Disease Type C1 Trial

OLE Trial Data Shows Arimoclomol Efficacy for NPC

Audrey’s Story: Life With Niemann-Pick Disease Type C

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

Trial of Niemann-Pick Disease Treatment is Expanded

FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug

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

Phase 1 Trial for Niemann-Pick Disease Type C Has Completed Enrollment

This Company Just Provided a Grant for a Niemann-Pick Disease Type C Screening Program

ICYMI: FDA Approves Expanded Access Application of Experimental Niemann-Pick Type C Drug

Metastatic Breast Cancer: Navigating Grief

Rethinking What It Means to Live With Acromegaly

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

Let’s Work Together!

Keep Up to Date

Learn More