16 July 2024 Leiden, The Netherlands, 16 July 2024 – Azafaros B.V. today announced positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic diagnosis of either GM2 gangliosidosis or Niemann-Pick disease type C (NPC). About the Trial The trial was conducted across three sites in Brazil, …
7th Annual DART To The Finish Charity Walk * Sat. * Sept. 28, 2024
Gather your friends and family and join is for another invigorating 2-mile walk with beautiful views of Long Island Sound around Tod’s Point in Old Greenwich, and help us raise much needed funds to find a cure for Niemann-Pick type C disease. No beach pass, no problem. DART has you covered! The morning always makes …
IntraBio files New Drug Application for N-acetyl-L-leucine (IB1001) for the treatment of NPC
IntraBio Inc. is pleased to share that the New Drug Application (NDA) for N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease type C (NPC) was submitted to the US Food and DrugAdministration (FDA) in January 2024. The FDA has a 60-day filing review period to determine whetherthe NDA is complete and accepted for review. With …
Azafaros’ Phase 2 RAINBOW study for nizubaglustat in NPC and GM2 patients fully enrolled
December 11, 2023 Leiden, The Netherlands, 11 December 2023 – Azafaros B.V. today announces that it has exceeded the target number of patients in the enrollment of its Phase 2 clinical trial, RAINBOW (NCT05758922), treating patients with severe rare metabolic diseases. The success in enrollment is a major milestone for Azafaros’ lead asset, nizubaglustat, which is …
IV Adrabetadex (cyclodextrin) Trial for Neonatal Liver Disease Started
Researchers from St. Louis Children’s Hospital at Washington University School of Medicine are enrolling infants 0-6 months with diagnosed NPC disease and evidence of liver disease. The purpose of the study is to learn how well adrabetadex can reduce liver damage in infants diagnosed with NPC disease. The study has an initial 6-week phase during …
2023 DART To The Finish charity walk raises over $150,000 for NPC research!
After a rainy morning, the skies cleared enough for over 150 supporters of DART to walk the 2-mile plus course around Tod’s Point in Old Greenwich to raise much needed funds for NPC research. Joining us this year was a group from The Prospector Theater in Ridgefield, CT where Andrew Marella works every week as …
IntraBio Completes Recruitment for IB1001 NPC Pivotal Clinical Trial
OXFORD, UK / December 08, 2022 / IntraBio Inc announced today that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 – NCT05163288). Enrolment for IB1001-301 commenced in September 2022. Patients were screened across 13 multinational trial sites …
DART to The Finish Charity Walk Raises $175,000
The fifth annual DART to The Finish charity walk benefitting Dana’s Angels Research Trust (DART) took place Saturday, September 24, 2022, at Greenwich Point Park in Old Greenwich, and virtually across the country. More than 300 participated and helpedraised more than $175,000, which will go towards supporting crucial research into better treatments, clinical trials and …
FDA Advisory Committee Votes To Recommend Arimoclomol as Effective Treatment for NPC
PDUFA Action Date for the Arimoclomol NDA is September 21, 2024 CELEBRATION, Fla., Aug. 02, 2024 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) …
Zevra Announces FDA Advisory Committee Meeting To Review Arimoclomol For The Treatment Of NPC
July 9, 2024 at 9:00 AM EDT Meeting with the recently formed GeMDAC set for August 2, 2024 CELEBRATION, Fla., July 09, 2024 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has indicated that it will convene a meeting with the recently formed Genetic …
Azafaros announces completion of Phase 2 RAINBOW study evaluating nizubaglustat in NPC and other rare disease patients
Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW (Phase 2 RAINBOW study NCT05758922). The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients older than 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The aim of the RAINBOW study is …