Cochrane at the WHO Executive Board 2024
Last week, Cochrane participated in the154th session of the World Health Organization (WHO) Executive Board meeting in Geneva.The Executive Board is composed of 34 technically qualified representatives from different Member States, elected for three-year terms. Every January, the Executive Board meets at WHO headquarters to discuss global public health priorities for the year ahead and set the agenda for theWorld Health Assembly.In addition to the Executive Board, all Member States and civil society organizations in official relations with WHO, such as Cochrane, are given the opportunity to contribute. This provided our de...
Source: Cochrane News and Events - February 1, 2024 Category: Information Technology Authors: Muriah Umoquit Source Type: news
Endpoint Protection in Rare Disease Trials: Safeguarding Data Integrity for Reliable Outcomes, Upcoming Webinar Hosted by Xtalks
In this webinar, gain insights into essential strategies for constructing a robust framework for endpoint protection in rare disease clinical trials. Attendees will learn strategies for ensuring quality clinical research organization (CRO) oversight of clinical data. The featured speaker... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 1, 2024 Category: Pharmaceuticals Tags: CCA FVT Source Type: news
Position statement on lung cysts, cystic lung disease
The U.K. Cystic Lung Disease Rare Disease Collaborative Network issued a position statement in January about cystic lung disease diagnosis.Simon Johnson, PhD, professor of respiratory medicine at the University of Nottingham, and colleagues highlighted that "in many cases a multidisciplinary diagnosis can be made without the need for lung biopsy" but that in some instances tissue sampling may be necessary when noninvasive methods leave diagnostic doubt. The position statement was prompted by rare cystic lung diseases increasingly recognized as a result of wider application of CT scanning. Johnson and colleagues acknowledge...
Source: AuntMinnie.com Headlines - January 19, 2024 Category: Radiology Authors: AuntMinnie.com staff writers Tags: Clinical News Source Type: news
Strategic Design Decisions in Rare Disease Trials, Upcoming Webinar Hosted by Xtalks
In this free webinar, gain insights into adapting endpoints for the needs of rare disease trials. Attendees will also learn strategies for conducting tailored rater training and targeted data quality monitoring. The featured speaker will also describe ways to evaluate clinical... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - January 17, 2024 Category: Pharmaceuticals Source Type: news
Vertex ’s CRISPR drug notches 2nd FDA approval for another rare disease
Just last month, the Boston drugmaker and its partner, CRISPR Therapeutics, earned approval to market the same drug to treat sickle cell disease. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 16, 2024 Category: Pharmaceuticals Authors: Hannah Green Source Type: news
mRNA technology could be possible treatment for rare diseases
By exploiting the technology used in COVID-19 vaccines, a team led by UCL, King's College London and Moderna scientists has created an effective therapy for a rare disease, in a study in mice, demonstrating the technology’s potential therapeutic use in people.
The research, published in Science Translational Medicine, found that messenger RNA (mRNA) could be used to correct a rare liver genetic disease known as argininosuccinic aciduria in a mouse model of the disease. (Source: World Pharma News)
Source: World Pharma News - January 11, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
FDA Grants Soligenix Fast Track Designation for Dusquetide in the Treatment of Oral Lesions of Beh çet's Disease
PRINCETON, N.J., Jan. 8, 2024 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - January 8, 2024 Category: Drugs & Pharmacology Source Type: news
Rare disease drug developer pays $6M after DoJ alleges 'kickback' scheme around lab tests
The payment will go to Medicare and Medicaid programs that the Justice Department alleges covered one of Ultragenyx's drugs; the company says its free-testing program saved those programs money. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 5, 2024 Category: Biotechnology Authors: Ron Leuty Source Type: news
A Roadmap to Better Rare Disease Clinical Trials: 3 Tips for Effective Engagement with Patient Advocacy Groups
In rare disease research, companies like IndoUSRare are exploring effective strategies such as establishing clinical research sites in countries with a higher incidence of specific rare diseases. This approach, coupled with the utilization of AI-enhanced tools for clinical trial... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - January 3, 2024 Category: Pharmaceuticals Tags: SVY NPT TRI Source Type: news
Lululemon ’s Founder Made A Fortune But Lost Out On Billions. Inside His Struggle To Move On
It’s been a decade since Chip Wilson left his yoga-inspired fashion brand amid controversy. The billionaire, who dumped most of his stake years ago, is still searching for a new legacy – backing another sportswear company, investing in real estate and racing to cure a rare disease. t’s just before…#chipwilson #newyorkcitys #westvillage #fifthavenue #wilson #fshd #nt #jerrylewis #als #lougherigs (Source: Reuters: Health)
Source: Reuters: Health - January 2, 2024 Category: Consumer Health News Source Type: news
Curiosity and Compassion Fuel Rare Disease Research
Lauren Drouin shares how personal connections and scientific curiosities drive her work on gene therapy viral vectors. (Source: The Scientist)
Source: The Scientist - January 1, 2024 Category: Science Tags: Magazine Issue Source Type: news
The Petri Dish: Boston biotech scores FDA approval, antiviral maker stops 3 studies
Chiesi Global Rare Diseases, a Boston-based business unit of Italian biopharma firm Chiesi Group, scored an approval from the U.S. Food and Drug Administration for a skin disorder drug. Plus, other life sciences news you may have missed. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - December 28, 2023 Category: Biotechnology Authors: Rowan Walrath 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
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. (NasdaqGS: ZVRA) a rare disease therapeutics company, today announced it resubmitted its New Drug Application (NDA) for arimoclomol, an investigational therapeutic... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - December 27, 2023 Category: Drugs & Pharmacology Source Type: news
Rare Diseases: Considerations for the Development of Drugs and Biological Products
(Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - December 22, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
