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Azafaros Media Release | March 12, 2024
Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients.
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Mar 14, 20241 min read
Zevra Therapeutics Media Release | March 4, 2024
Zevra Therapeutics Provides FDA Update On The PDUFA Action Date For Arimoclomol As A Treatment For Niemann-Pick Disease Type C Read the...
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Mar 5, 20241 min read
IntraBio Media Release | February 7, 2024
The New England Journal of Medicine has published a "Science behind the study" editorial on IB1001 Read the full announcement from...
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Feb 8, 20241 min read
IntraBio Media Release | February 7, 2024
The New England Journal of Medicine has published the detailed results of the IB1001-301 Phase 3, Pivotal study with N-acetyl-L-leucine...
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Feb 3, 20241 min read
IntraBio Community Update | February 1, 2024
New Drug Application for IB1001 for the treatment of NP-C disease was submitted to the US FDA in January 2024 Read the full statement...
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Feb 1, 20241 min read
Zevra Therapeutics Media Release | January 8, 2024
Zevra Therapeutics Receives FDA Acceptance Of Resubmission Of NDA For Arimoclomol As A Treatment For Niemann-Pick Disease Type C Read the...
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Jan 10, 20241 min read
Zevra Therapeutics Media Release | December 27, 2023
Zevra Therapeutics Announces Resubmission Of Arimoclomol New Drug Application To The U.S. Food And Drug Administration Read the full...
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Dec 28, 20231 min read
Azafaros Media Release | December 11, 2023
Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled Read the full statement from...
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Dec 21, 20231 min read
Cyclo Therapeutics Media Release | December 18, 2023
Cyclo Therapeutics Announces Positive Outcome from Type C Meeting with the U.S. FDA Discussing Trappsol® Cyclo™ Clinical Program for the...
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Dec 20, 20231 min read
Meet and Greet with Cyclo Therapeutics
The Australian NPC Disease Foundation hosted a meet and greet with Cyclo Therapeutics on Zoom on 12th December.
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Dec 13, 20231 min read
Zevra Therapeutics Media Release | November 20, 2023
Zevra announces the completion of its acquisition of Acer Therapeutics. Zevra states they are committed to the arimoclomol expanded...
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Nov 21, 20231 min read
Kisbee Therapeutics and Zevra Therapeutics Updates
"We remain steadfast in our key priorities to the NPC community," - Zevra Therapeutics Read the full statement from Zevra Therapeutics...
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Sep 11, 20231 min read
Intrabio Media Release | June 29, 2023
"IntraBio Announces Positive Pivotal Trial Results of IB1001 for the Treatment of Niemann-Pick Disease Type C" Read the full article...
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Jun 29, 20231 min read
Azafaros Media Releases | January 5-9, 2023
"Azafaros Receives FDA's IND Clearance and Fast Track Designation for Lead Asset AZ-3102" Read the press release from January 5, 2023...
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Jan 10, 20231 min read
Intrabio Media Release | December 5, 2022
"IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on...
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Dec 6, 20221 min read
Pfrieger's Digest | Issue 7 - September 2022
Pfrieger's Digest for Niemann-Pick type C: Summaries of research advances on selected peer-reviewed publications in scientific journals....
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Oct 10, 20221 min read
It's a wrap! 2022 NPC Conference
The annual NPC Conference, both live and online, attended by patients, families, clinicians and researchers of Niemann-Pick disease.
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Jun 30, 20222 min read
Pfrieger's Digest | Issue 6 - May 2022
Pfrieger's Digest for Niemann-Pick type C: Summaries of research advances on selected peer-reviewed publications in scientific journals....
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Jun 6, 20221 min read
Azafaros Media Release | March 24, 2022
"Second Orphan Drug Designation Supports Azafaros’ Strategy to Develop AZ-3102 as a Disease Modifying Treatment in a Range of Severe Rare...
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Mar 25, 20221 min read
Orphazyme Media Release | March 22, 2022
"Orphazyme announces withdrawal of European Marketing Authorisation Application for arimoclomol for the treatment of Niemann-Pick disease...
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Mar 25, 20221 min read