Message from MIHRA Leadership to Welcome 2026!
✨Happy New Year✨ to all!
To our myositis community of patients, patient research partners, clinicians, scientists, trainees, regulatory, advocacy and industry partners – wishing you and your family the best of ✨peace, ✨joy and ✨health for this coming year.
Artist: Nancy Willis
✨The Myositis Community Standing Fast
While 2025 ushered in unforeseen challenges for sound science, patients and the welfare of our colleagues in government positions, the MIHRA community held steadfast to hope and to focus on rare and myositis disease progress as well as the scientific relevance of the authentic patient voice.
Thus as 2026 begins to unfold, the MIHRA community remains illuminated by the strength, compassion, generosity and innovations of individuals and partner organizations. MIHRA’s ✨IIM Specialty Clinics (ISC) continues to expand globally and over a hundred sites now committed to ensuring trial site integrity, efficiency and optimization through MIHRA’s ✨Clinical Trial Sites Network (CTSN).
In response to the cancelled FDA–NIH Rare Disease Day conference scheduled for February 27–28, 2025; MIHRA, in partnership with The Myositis Association (TMA) and Nori’s Fight, rapidly pivoted within a week to a live virtual We Care for Rare Summit. The summit gathered >400 registrants worldwide to face these rough waters with a firm message: “we stand for science” and “we stand for science in rare diseases”. Among the MIHRA community’s major advancements (mitochondrial biomarkers, exercise as medicine, improved composite index) shared was the work of ✨The MIHRA Heavy Lifters✨.
✨The MIHRA Heavy Lifters✨ are lifelong high-performing athletes living with IIMs. Their work delineates both the patient and scientific perspectives on a critical barrier in IIM care and research: the potentially unjustified reliance on the MMT8 to detect moderate to severe muscle impairment especially when compared to validated muscle performance measures, like the Functional Index-3 (FI-3). They issued two practical clinical guides for: (i) ✨Clinicians to Recognize the Likelihood of Active Muscle Disease When Traditional Measures are Indeterminate, (ii) ✨Shared Decision-Making (SDM) to Help Prevent Delayed Treatment of Active Muscle Disease.
✨Standing Fast with MIHRA PATH
2025 heralded a major breakthrough for the myositis community! MIHRA Partnership to Advance Therapeutic Horizons (PATH) launched the myositis community’s formal public–private partnership (PPP) with the U.S. Food and Drug Administration (FDA) through its Center for Drug Evaluation and Research (CDER) to accelerate therapeutic breakthroughs in myositis diseases through clinical trial readiness and novel approaches to trial design. MIHRA PATH is a pre-competitive multi-stakeholder research collaboration whereby regulatory & government scientists, patient experts, academic investigators & centers, industry partners, advocacy groups and representation from organizations like the American College of Rheumatology (ACR) work side-by-side to target the most pressing barriers to clinical trial readiness in myositis diseases.
MIHRA PATH carries on MIHRA’s current work and strives to achieve regulatory standards at each step. This includes clear guidance on trial design elements (trial structures and optimization techniques, flare, remission, response, eligibility, patient engagement and novel analytic applications), validation of higher performing endpoints for muscle performance, skin and inclusion of sentinel organ involvement in the hopes that myositis diseases might finally achieve a fully validated total composite score in non-IBM myositis diseases. MIHRA PATH also ensures quality and regulatory standard guardrails in the production of our planned patient-reported outcomes research.
Establishing multi-stakeholder community consensus on how to integrate the controversial diagnosis of ‘polymyositis’ into eligibility criteria is a pivotal aspect of sound trial design in myositis diseases. We anticipate this work to be completed and available for clinical integration by August 2026.
In November 2025, MIHRA PATH’s Endpoint Training & Certification Program (ETC) was presented during the myositis research meeting at the ACR Annual Scientific Conference. supplying a definitive answer to several presentations which reported the difficulties and lack of investigator confidence in implementing these critical endpoints. MIHRA PATH ETC is a rigorous and responsive program developed with instrument creators to ensure (a) correct implementation of endpoints across sites and studies,(b) standardize trial & natural history data collection at a high level of integrity, & (c) rights protection for instrument creators and their intellectual contributions.
✨Standing Fast with Global Innovations
MIHRA Patient Priorities in Research is a major innovation in rare diseases introduced by the MIHRA Patient Advisory and MIHRA Board of Directors to ensure that patient voice is central, operational, traceable, and honored. The program is a standing investigation using qualitative and mixed methods approaches to assert the patient voice as influential in research and funding selection. The 2025 results have informed MIHRA’s 2026 Career Enhancement Awards and will continue to strengthen MIHRA’s readiness for our future grant-making and community investment. The 2025 results anticipate publication submission for May 2026. MIHRA hopes this endeavor will influence research prioritization worldwide.
2026 will launch MIHRA Foundation’s alliance with Global Genes RARE-X for the myositis community’s patient-driven and generated database with highly detailed patient-relevant content comprised of symptoms and ‘exposome’ data. RARE X’s remarkable interface will stream case-by-case patient-provided data with MIHRA’s Prospective Registry. This intersection of exquisitely detailed patient-provided information with the high-quality standardized data ensured by MIHRA PATH Endpoint Training & Certification is anticipated to accelerate natural history discoveries in these rare diseases.
✨Standing Fast with the Future of Myositis Research
MIHRA’s Career Enhancement Program engages emerging leaders and all-comers interested in myositis research or care through structured programs. In that spirit, MIHRA progressively strengthens their educational offerings and opportunities. Multi-disciplinary, specialty and patient-inclusion programming, – such as MIHRA Scientific Seminars, Community Forums, Journal Clubs and Global Case Conferences – continue to stretch the boundaries of multi-stakeholder knowledge, exchange of insight and collaboration that advances research and care in myositis diseases.
The MIHRA community is proud to announces ✨nine✨ recipients of the MIHRA Professor Paul H. Plotz Career Enhancement Award to GCOM 2026. This cohort reflects MIHRA’s values in collegial and collaborative attributes as well as a broad diversity of career level, geographical practice, discipline, specialty (including two pediatric investigators), and research interests strongly aligned with patient-voiced research priorities. MIHRA ensures a rigorous review process which each application initially reviewed by up to 7 reviewers with at least one patient partner, one rehabilitation specialist and two physicians. We very much look forward to celebrating these candidates at GCOM 2026!
January 2026 rings in the initiation of MIHRA intensive hands-on knowledge & skills-based workshops! The first course focuses on scientific methods and software applications concerned with authenticity & inclusion of the patient voice, the multi-stakeholder voice and the development of patient-reported instruments. Research methodologies regarding patient-reported experience, qualitative, mixed methods, survey and outcome measure development in rare diseases are a critical integration in the advancement of rare diseases. However, these complex sciences are at risk of diminished scientific quality if investigators lack training & apprenticeship in these applications. These trainings ensure such projects are: (a) met with appropriate scientific rigor, (b) provide apprentice opportunities for novices and (c) accelerate assembly of strongly skilled collaborative teams that efficiently meet these research needs with high scientific merit.
Thank you for being part of ✨this year and the year to come✨ whether you attended a session, mentored someone quietly, shared lived experience, reviewed a draft, supported MIHRA materially, or simply kept showing up.
In 2026, may we continue to create a world where we can cure myositis.
With gratitude,
The MIHRA Board of Directors
Barbara Shafranski, Elie Naddaf, Floranne Ernste, Helene Alexanderson, Julie Paik, Lesley Ann Saketkoo, Mazen Dimachkie, Pedro Machado
MIHRA Patient Advisory
Anneli Dihkan, Chip Galloway, Christine Lowe, Ingrid de Groot, Irene Oakley, Joe Sanchez, Karen Cheng, Roland Mischke, Silke Schlüter, Suzanne Edison
MIHRA Scientific Advisory
Annet van Royen, Bianca Lang, Chet Oddis, David Isenberg, Fred Miller, Ingrid Lundberg, Jiri Vencovsky, Kristin Highland, Lisa Christopher-Stine, Marianne de Visser, Masataka Kuwana, Vicky Werth