Niemann-Pick Type C – 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…

“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…

stevepb / Pixabay

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

Post author:Scott Carlson
Post published:June 11, 2019
Post category:Niemann-Pick Disease

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.…

Source: Pixabay

Family Sues Biotech Companies For Privacy Breach

Post author:Meagan Fulps
Post published:January 22, 2018
Post category:Niemann-Pick Disease Rare Disease

The Hempel family decided to take action against Vtesse and Sucampo Phamaceuticals who allegedly stole and sold their private information for profit, reported Forbes. The Hempel family has two twin…

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

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

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

Family Sues Biotech Companies For Privacy Breach

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