/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-06 15:42:412025-02-06 15:42:41Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for the Treatment of Niemann-Pick Disease Type C — Presenting a Rescored 4-domain NPC Clinical Severity Scale
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 11:37:542025-02-03 11:52:43Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-domain Niemann-Pick Disease Type C (NPC) Clinical Severity Scale (5DNPCCSS) And Analysis in an NPC Clinical Trial Data Set
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 11:36:262025-02-03 11:52:51Arimoclomol Safety Profile in the Treatment of Niemann-pick Disease Type C In a Real-world Setting: Long-term Safety Data from an Expanded Access Program in the United States
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 11:33:002025-02-03 11:52:55Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann-Pick Type C – Data From 48 Months Open Label Trial
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 11:00:242025-02-03 12:02:48Safety of Arimoclomol in a Pediatric Sub-Study of Niemann-pick Disease Type C Patients Aged 6 To <24 Months at Study Enrollment
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 10:57:532025-02-03 12:02:53Perseverance Is Key for Regulatory Success in Ultra-Rare Diseases – Key Learnings from Arimoclomol’s Regulatory Journey
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 10:54:142025-02-03 12:03:00Arimoclomol for the Treatment of Niemann-pick Disease Type C In a Real-world Setting: Long-term Data from an Expanded Access Program in the United States
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 10:51:412025-02-03 12:03:07Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 10:44:052025-02-03 12:03:13Efficacy Results from A 12-month Double-blind Randomised Trial of Arimoclomol for Treatment of Niemann-Pick Disease Type C – Presenting A Rescored 4-domain NPC Clinical Severity Scale
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2024-11-11 10:45:062025-02-03 12:03:28Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-Domain Niemann-Pick disease type C (NPC) Clinical Severity Scale (5DNPCCSS)
We may request cookies to be set on your device. We use cookies to let us know when you visit our websites, how you interact with us, to enrich your user experience, and to customize your relationship with our website.
Click on the different category headings to find out more. You can also change some of your preferences. Note that blocking some types of cookies may impact your experience on our websites and the services we are able to offer.
Essential Website Cookies
These cookies are strictly necessary to provide you with services available through our website and to use some of its features.
Because these cookies are strictly necessary to deliver the website, refusing them will have impact how our site functions. You always can block or delete cookies by changing your browser settings and force blocking all cookies on this website. But this will always prompt you to accept/refuse cookies when revisiting our site.
We fully respect if you want to refuse cookies but to avoid asking you again and again kindly allow us to store a cookie for that. You are free to opt out any time or opt in for other cookies to get a better experience. If you refuse cookies we will remove all set cookies in our domain.
We provide you with a list of stored cookies on your computer in our domain so you can check what we stored. Due to security reasons we are not able to show or modify cookies from other domains. You can check these in your browser security settings.
Google Analytics Cookies
These cookies collect information that is used either in aggregate form to help us understand how our website is being used or how effective our marketing campaigns are, or to help us customize our website and application for you in order to enhance your experience.
If you do not want that we track your visit to our site you can disable tracking in your browser here:
Other external services
We also use different external services like Google Webfonts, Google Maps, and external Video providers. Since these providers may collect personal data like your IP address we allow you to block them here. Please be aware that this might heavily reduce the functionality and appearance of our site. Changes will take effect once you reload the page.
Google Webfont Settings:
Google Map Settings:
Google reCaptcha Settings:
Vimeo and Youtube video embeds:
Privacy Policy
You can read about our cookies and privacy settings in detail on our Privacy Policy Page.