global epidemiological and genetic burden of rare neurological diseases in children

Original title: “Global epidemiological and genetic burden of rare neurological diseases in children.”

international recommendations for child to adult care transition in rare neurological diseases a scoping review and delphi consensus study

Original title: “International recommendations for child to adult care transition in rare neurological diseases: A scoping review and Delphi consensus study.”

creating shared vision in patient partnered research a case example of patient engagement from the pave project producing an arthritis value framework with economic evidence paving the way for rare childhood disease

Original title: “Creating shared vision in patient partnered research: a case example of patient engagement from the PAVE project (producing an arthritis value framework with economic evidence: paving the way for rare childhood disease).”

The key issues for industry and FDA as the Orphan Drug Act hits its 40th anniversary. The key issues for industry and FDA as the Orphan Drug Act hits its 40th anniversary. ... As patients and the public health community mark the 40th anniversary of the Orphan Drug Act of 1983, biopharma companies and FDA officials are acknowledging continued challenges in testing, approving, and producing new treatments for critical conditions affecting small patient populations. And recent legislation has established an initiative to accelerate the development of more effective treatments for amyotrophic lateral sclerosis (ALS) through an FDA Rare Neurodegenerative Disease grants program].5 The awards aim to cover R&D costs of new therapies for ALS and a range of rare neurogenerative diseases in adults and children, as spelled out in an FDA action plan issued in June 2022. Efforts to advance rare disease treatments, however, may be stymied by the ongoing legal battle that has raised challenges to FDA policies governing the scope of orphan drug exclusivity policy. Despite that court ruling, FDA has insisted that it will continue to tie orphan drug exclusivity only to approved uses or indications.6 The agency has pressed Congress to enact legislation to clarify this stance, but so far has failed to gain action on Capitol Hill. The program has led to market approval of more than 600 therapies to treat serious diseases affecting less than 200,000 patients in the US, investment spurred by grants and tax credits to help fund very small clinical trials, exemptions from hefty user fees, plus seven years added of marketing exclusivity for approved orphans and more incentives for developing rare disease treatments for children.1 · FDA notes that orphan therapies accounted for more than half of novel new drugs approved in 2022, and that the agency already has approved two notable rare disease treatments this year.

Original title: “Kudos and Hurdles in Tackling Rare Diseases | Applied Clinical Trials Online”

According to IMARC Group the global virtual clinical trials market size reached USD 252 5 Million in 2025 and is projected to reach USD 379 0 Million by 2034 expanding at a CAGR of 4 48 during 2026 2034 This ... Press release - IMARC Group - Virtual Clinical Trials Market Size, Share, In-Depth Analysis, Trends and Forecast 2026-2034 - published on openPR.com Advances in targeted therapies, immunotherapies, and precision medicine are reshaping cancer care and pushing sponsors toward trial designs that can accommodate more individualized treatment protocols. The growing understanding of tumor genetics is also expanding demand for niche trials targeting rare and genetically defined cancers, where virtual recruitment models help overcome the patient-scarcity challenges that limit traditional site-based enrollment. This steady growth reflects the deepening integration of advanced imaging technology into veterinary practice, driven by rising pet humanization, escalating chronic disease burden in animals, and the rapid proliferation of specialized veterinary hospitals and clinics globally.

Original title: “Virtual Clinical Trials Market Size, Share, In-Depth Analysis, Trends and Forecast 2026-2034”

On September 14, a bipartisan coalition of Representatives introduced the Priority for Pediatric Cures Act, which would make the Rare Pediatric Disease Priority Review Voucher (PRV) Program permanent.

Original title: “Bipartisan Bill Aims to Make the Rare Pediatric Disease PRV Program Permanent”