Treatment With N-Acetyl-L-Leucine Beneficial for Niemann-Pick Disease
THURSDAY, Feb. 1, 2024 -- Treatment with N-acetyl-L-leucine (NALL) for 12 weeks yields better neurological status than placebo among patients with genetically confirmed Niemann-Pick disease type C, according to a study published in the Feb. 1 issue... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - February 1, 2024 Category: Pharmaceuticals Source Type: news

Potentially Protective Agent Shows Benefit in Niemann-Pick Type C
(MedPage Today) -- Patients with Niemann-Pick disease type C who received N-acetyl-L-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, showed reduced neurologic signs and symptoms after 3 months, a phase... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - January 31, 2024 Category: Neurology Source Type: news

FDA Acceptance of Zevra's Arimoclomol NDA Filing for Niemann-Pick Disease Type C (NPC) Results in XOMA Making a $1 Million Milestone Payment to LadRx
EMERYVILLE, Calif., Jan. 11, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation XOMA, the biotech royalty aggregator, announced today that based upon the U.S. Food and Drug Administration's (FDA) acceptance of Zevra Therapeutics' New Drug Application (NDA) for arimoclomol, an orally-delivered,…#emeryville #xomacorporationxoma #fda #nda #npc #xoma #ladrx #arimoclomol #bradsitko #zevra (Source: Reuters: Health)
Source: Reuters: Health - January 11, 2024 Category: Consumer Health News Source Type: news

Zevra Therapeutics Announces Resubmission of Arimoclomol New Drug Application to the U.S. Food and Drug Administration
CELEBRATION, Fla., Dec. 27, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. ZVRA a rare disease therapeutics company, today announced it resubmitted its New Drug Application (NDA) for arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC) to…#celebration #fla #zevratherapeuticsinc #newdrugapplication #nda #npc #fda #pdufa #zevra #neilmcfarlane (Source: Reuters: Health)
Source: Reuters: Health - December 27, 2023 Category: Consumer Health News Source Type: news

Citizen Scientists Are Driving Medical Research. Now They Need A Constitution
More than a dozen people showed up in November, 2007, at the National Institutes of Health (NIH) lab run by Christopher Austin. The parents of children dying from the lethal cholesterol metabolism disorder, Niemann-Pick disease type C (NPC), gathered alongside scientists and doctors looking for a treatment. They wanted to work together to find effective drugs and get them into clinical trials within a few years, accelerating the typical timeline for drug development. They hoped to find a compound that might extend the lives of children with the rare disease, who typically died by age 19. In terms of the science, the meetin...
Source: TIME: Health - April 14, 2023 Category: Consumer Health News Authors: Amy Dockser Marcus Tags: Uncategorized freelance health Source Type: news

What Are Causes of Childhood Interstital Lung Disease?
Discussion Interstitial lung disease in children (chILD) is less understood than adult interstitial lung disease (ILD) and is rare. Prevalence is ~ 0.13 to 16.2 per 100,000 children under age 17 years. chILD is an general term for respiratory disorders that are heterogeneous, chronic and impair lung function. While some define diffuse parenchymal lung disease (DPLD) separately, the term ILD usually encompases DPLD. chILD has variable definitions and is “usually diagnosed if three of the following features are present: 1) respiratory symptoms (cough, rapid and/or difficult breathing, and exercise intolerance), 2) res...
Source: PediatricEducation.org - August 1, 2022 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news

Endpoint Considerations to Facilitate Drug Development for Niemann-Pick Type C (NPC): Key Themes and Future Directions from the January 2022 Public Workshop - 08/04/2022
Endpoint Considerations to Facilitate Drug Development for Niemann-Pick Type C (NPC): Key Themes and Future Directions from the January 2022 Public Workshop (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 21, 2022 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Orphazyme Provides Update for Planned NDA Resubmission for Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the United States
Copenhagen, Denmark, February 11, 2022 – Orphazyme A/S (ORPHA.CO; ORPH) (“Orphazyme” or the“Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - February 11, 2022 Category: Drugs & Pharmacology Source Type: news

Endpoint Considerations to Facilitate Drug Development for Niemann-Pick Type C (NPC) Public Workshop - 01/24/2022 - 01/25/2022
Endpoint Considerations to Facilitate Drug Development for Niemann-Pick Type C (NPC) Public Workshop (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - December 3, 2021 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Orphazyme Provides Regulatory Update from FDA on Arimoclomol for Niemann-Pick Disease Type C
Copenhagen – June 18, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases, today announced it has received a... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - June 18, 2021 Category: Drugs & Pharmacology Source Type: news

Polaryx Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Niemann Pick Disease Types A and B With PLX-300
PARAMUS, N.J., Dec. 18, 2020 -- (Healthcare Sales & Marketing Network) -- Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received from the U.S. Food and Dru... Biopharmaceuticals, Neurology, FDA Polaryx Therapeutics, Niemann Pick Disease (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - December 18, 2020 Category: Pharmaceuticals Source Type: news

Orphazyme Announces FDA Acceptance and Priority Review of New Drug Application for Arimoclomol for Niemann-Pick Disease Type C
Copenhagen, Denmark, September 16, 2020– Orphazyme A/S (ORPHA.CO), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announced that the U.S.... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - September 16, 2020 Category: Drugs & Pharmacology Source Type: news

Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease
NIH observational study suggests that the drug may decrease risk of pneumonia and death in this population. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - September 9, 2020 Category: American Health Source Type: news

Orphazyme Completes Rolling Submission of New Drug Application To U.S. FDA for Arimoclomol for Niemann-Pick Disease Type C
Copenhagen, Denmark, July 20, 2020– Orphazyme A/S (ORPHA.CO), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response in order to develop and commercialize novel therapeutics for the treatment of... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - July 20, 2020 Category: Drugs & Pharmacology Source Type: news