"Second Orphan Drug Designation Supports Azafaros’ Strategy to Develop AZ-3102 as a Disease Modifying Treatment in a Range of Severe Rare Inherited Metabolic Disorders" Read the full article here: Article Link Announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102 Based on its mode of action, AZ-3102 has broad applicability in addressing these inherited metabolic disorders. "As we explore the broad potential of AZ-3102 a

"Orphazyme announces withdrawal of European Marketing Authorisation Application for arimoclomol for the treatment of Niemann-Pick disease type C" Read the full article here: Article Link Decision to withdraw the European Marketing Authorisation Application comes ahead of scheduled final vote on the application later this month Orphazyme intends to request Type C Meeting with the U.S. Food and Drug Administration to discuss potential pathway for resubmission of the New Drug Ap

The second NPC Charity Gala was held February 26, 2022 - close to 2 years after the original date in Oct 2020. It was a great success, raising a little over $50,000 net for research conducted at The Florey Institute of Neuroscience and Mental Health. For an event that many believed would barely break-even, we are so thrilled to have delivered on a truly engaging night that raised fantastic awareness and funds for research on NP-C treatments. The 2022 NPC Charity Gala faced ma