International Niemann–Pick Disease Alliance
12 December 2020 Orphazyme Statement to the NPC Community Dear Global Community of NPC Patients, Families and Collaborators: You may have seen the news announced on 10 December that Kim Stratton has decided to resign as CEO of Orphazyme...
Transaction Includes Uplisting to Nasdaq and Reverse Stock Split Gainesville, FL – (Businesswire) –December 9, 2020 – Cyclo Therapeutics, Inc. (“Cyclo Therapeutics” or the “Company”) (NasdaqCM:CYTH, CYTHW), a clinical stage biotechnology company developing a cyclodextrin platform for the treatment...
The INPDR releases its ambitious Strategic Business Plan 2020 – 2022 setting targets to continue leading edge work and provide impetus for the future. The INPDR, a ground-breaking patient-focused rare disease-specific registry, has announced plans to build on progress...
Conference Brings Together Medical Researchers and Health Professionals to Exchange Information on Lysosomal Diseases Gainesville, FL – (Businesswire) – 19 November 2020 Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical stage biotechnology company developing a cyclodextrin platform for the treatment...
Orphazyme Submits European Marketing Authorisation Application for Arimocolmol for Treatment of Niemann-Pick disease type C Follows U.S. Food and Drug Administration (FDA) acceptance of New Drug Application (NDA) with Priority Review in September 2020 · Regulatory decisions expected in 2021...
Cyclo Therapeutics will now proceed in the US with its Phase 3 clinical trial of Trappsol® Cyclo™ for treatment of Niemann-Pick Disease Type C GAINESVILLE, FL – (Businesswire) – 21 October 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a...
– IB1001 demonstrated statistically significant and clinically meaningful change in both the primary and secondary endpoints – Significant improvement in gait, fine motor skills, speech, cognition, overall functioning and quality of life reported – IB1001 rapidly improved both motor...
“Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...
IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C Positive results reported from IntraBio’s multinational clinical trial with IB1001 for treatment of Niemann-Pick disease Type C (NPC) Statistically significant and clinically...
If approved, arimoclomol would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC) FDA has set target PDUFA action date of March 17, 2021 Copenhagen, Denmark, September 16, 2020 – Orphazyme A/S (ORPHA.CO), a...