viernes, 13 de noviembre de 2026
Advancing Clinical Trials for Rare and Ultra-Rare Diseases
Advancing Clinical Trials for Rare and Ultra-Rare Diseases
RealiseD is set to generate a novel comprehensive methodological and operational approach to design and conduct clinical trials for rare and ultra-rare diseases.
https://realised-ihi.eu/
Patient Survey
The IHI RealiseD project, to which EURORDIS has contributed, launched its Patient-Centred Survey on Clinical Trials in October. The survey aims to better understand the needs of people living with rare diseases, their families and carers, as well as the barriers they face when participating in clinical trials. By sharing their experiences, respondents can help make rare disease clinical research more accessible, inclusive and patient-centred. Take the survey now.
https://survey.erknet.org/#/s/kjcpbfqqtr
Europe has spent decades building its rare disease ecosystem. Why does it still need a plan? November 2026
Europe has spent decades building its rare disease ecosystem. Why does it still need a plan?
November 2026
Over the past two decades, the EU has made considerable progress on rare diseases, establishing specialist healthcare networks, supporting cross-border research programmes, developing shared information systems and introducing incentives for orphan medicine development.
Yet despite these achievements, millions of Europeans living with rare diseases continue to face lengthy diagnostic delays, fragmented care and unequal access to treatment.
The challenge is no longer simply to develop new initiatives, but to ensure that Europe’s existing efforts work together more effectively to address these persistent gaps.
https://www.eurordis.org/questions-on-european-blueprint/
In October, following 10 months of co-development with people living with rare diseases and stakeholders from across the rare disease community, EURORDIS-Rare Diseases Europe launched its European Blueprint for Rare Diseases.
The Blueprint sets out a shared vision for a more coordinated European response to rare diseases, proposing concrete priorities and actions to shape a future EU Action Plan on Rare Diseases.
But what exactly does the Blueprint propose, why is an EU Action Plan needed, and how can we turn this shared vision into reality?
Our latest Q&A article answers five key questions about the Blueprint, including how a future EU Action Plan could build on existing European and global initiatives.
UCB Study Highlights the Burden of Ocular Myasthenia Gravis by Chad Birt| Published on: Nov 12, 2026
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
by Chad Birt| Published on: Nov 12, 2026
Biopharmaceutical company UCB recently shared findings from a mixed-methods study examining the experiences and unmet needs of people living with ocular myasthenia gravis (oMG). The findings were presented at the 2026 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and Myasthenia Gravis Foundation of America (MGFA) Scientific Session.
Anna Kole, Global Patient Engagement Lead for Myasthenia Gravis at UCB, discusses the findings and explains how patient perspectives could improve clinical research and care.
https://checkrare.com/ucb-study-highlights-the-burden-of-ocular-myasthenia-gravis/
FDA Approves Pimjetmi (Pimicotinib) for Patients With Tenosynovial Giant Cell Tumor by Madaline Spencer| Published on: Nov 12, 2026
FDA Approves Pimjetmi (Pimicotinib) for Patients With Tenosynovial Giant Cell Tumor
by Madaline Spencer| Published on: Nov 12, 2026
The US Food and Drug Administration (FDA) has approved Pimjetmi (pimicotinib) as a systemic treatment for adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection may cause worsening functional limitation or severe morbidity.
https://checkrare.com/fda-approves-pimjetmi-pimicotinib-for-patients-with-tenosynovial-giant-cell-tumor/
PHINDER Analysis Highlights the Need for Earlier Pulmonary Hypertension Screening in Interstitial Lung Disease by Chad Birt| Published on: Nov 13, 2026
PHINDER Analysis Highlights the Need for Earlier Pulmonary Hypertension Screening in Interstitial Lung Disease
by Chad Birt| Published on: Nov 13, 2026
Parth Rali, MD, MBBS, FCCP, pulmonologist at Temple Health, discusses interim findings from the PHINDER study evaluating pulmonary hypertension (PH) screening strategies in patients with interstitial lung disease (ILD).
https://checkrare.com/phinder-analysis-highlights-the-need-for-earlier-pulmonary-hypertension-screening-in-interstitial-lung-disease/
Suscribirse a:
Entradas (Atom)
.png)
