Plain-language LGMD guide for families and non-specialist clinicians, including the 2I/R9 subtype. Developed by TREAT-NMD and Sarepta Therapeutics with international clinician and advocacy contributors.
Practical Support
Resources
Find organizations, registry information, emergency-preparation tools, and school or work resources to discuss with your local support team. Check the country and eligibility details before applying for a service.
FamiliesRegistryEmergency prepSchool and work
Start Here
Starting points for support and information
The Newcastle registry team's overview links to the Global FKRP Registry and explains how registry data support research. Ask the team about current registration and consent requirements.
One-on-one help with disease information, care centers, community referrals, and MDA programs. MDA services are available only in the U.S.; the team may suggest organizations elsewhere.
U.S.-focused assistance for some medication, insurance, travel, and caregiver costs. Availability depends on the specific program, eligibility, and funding; this link does not confirm an FKRP-specific fund.
By Situation
Use the resource that matches the practical problem in front of you
Preparation tools
Blank printable worksheets
Organize appointment, laboratory, emergency-summary, and school-support conversations without submitting or saving health information.
Emergency preparation
Keep clinician-written instructions current
Use a neutral preparation guide and blank summary without copying a generic treatment protocol.
Emergency
Emergency Prep
MDA links to disaster-preparation toolkits, checklists, and U.S. recovery services. Use these to plan logistics with your support network, and ask your clinical team for an individualized medical emergency plan.
School
School Support
MDA provides school-planning guides, information about U.S. IEP and 504 plans, and an LGMD educator flyer. Use these to prepare discussions with your child's education team; eligibility and procedures depend on the local school system.
Work
Work and Adjustments
Muscular Dystrophy UK explains workplace adjustments and provides a Workplace Adjustment Passport. Its legal and funding information is UK-specific, and individual schemes have their own eligibility rules.
Registry
Research Participation
Contact the Newcastle registry team about joining the Global FKRP Registry, the information collected, and consent. Participation supports research; it does not provide treatment or guarantee entry into a clinical trial.
Geography
What is broadly useful versus country-specific
International
- The TREAT-NMD family guide was developed for an international LGMD audience and includes 2I/R9.
- The Global FKRP Registry is international and coordinated from Newcastle. Confirm current registration and consent requirements with its team.
- Travel, insurance, and school systems still vary, so local adaptation matters.
United States
- MDA services are available only in the U.S.; its Resource Center may suggest local organizations for people elsewhere.
- MDA's school and disaster-preparation pages use U.S. systems and services.
- NORD assistance depends on each program's diagnosis, residency, financial, and funding requirements. Ask whether a relevant program is open.
United Kingdom
- Muscular Dystrophy UK provides workplace-adjustment resources and downloadable alert cards, including an LGMD card.
- Work-related rights and funding information are UK-specific; check each scheme's geographic coverage.
- Discuss the relevant alert card with your clinical team and check physical-card delivery availability with the organization.
How To Use This Page
Practical rules for keeping support information useful
Use this page for navigation, preparation, and logistics. Use the clinical pages for surveillance and disease explanation.
Keep local copies of emergency information, medication lists, equipment details, and specialist contacts.
Expect that school, work, travel, and benefits support will differ by country even when the diagnosis language is similar.
Registry participation and support organizations can help with navigation, but they do not replace individualized medical advice.
Selected Sources
Reference trail for this page
Official guide landing page confirms the audience, included LGMD subtypes, and development partners.
Registry operator's overview, with the Global FKRP Registry link and explanation of research uses.
Official contact details for FKRP registration, access, and consent questions.
Research-database description associated with the December 2023 ethics opinion, covering purpose, consent, and data governance; not a current recruitment notice.
One-on-one information and referrals. The page explicitly limits MDA services to the U.S.
Disaster-preparation toolkits, checklists, and U.S. emergency-recovery services.
School-planning materials, U.S. IEP and 504 information, and an LGMD educator flyer.
UK charity's physical-card order form and downloadable condition-specific cards, including LGMD.
Workplace-adjustment information and a downloadable passport. The source lists June 2025 as its own review date.
Program directory and eligibility information. Funding and availability vary; no FKRP-specific coverage is asserted here.
Continue Reading
Use these routes alongside the support links
Audience Guide
For Families
Plain-language entry page for building the right questions before appointments.
Core Reference
Monitoring and Care
Surveillance and multidisciplinary care framework for heart, lungs, sleep, mobility, and follow-up planning.
Evidence Layer
Trials and Studies
Registry and study records that connect support pages to current field activity.
Field Update
Therapeutic Pipeline
Dated status page for current FKRP-targeted programs and how to interpret sponsor or trial updates.