List of Resources For Rare Disease Patients In Texas
Texas families navigating rare disease diagnoses face complex challenges—but you're not alone. 30 million Americans live with rare diseases, and comprehensive support networks exist across the Lone Star State to help you find treatment options, financial assistance, and specialized care. Whether you just received a diagnosis or you're exploring personalized therapeutic options, this guide provides actionable resources that Texas patients can access immediately.
1. McGovern Medical School NORD Rare Disease Center of Excellence
McGovern Medical School at UTHealth Houston operates as a designated NORD Center of Excellence, providing comprehensive care for Texas patients with rare genetic conditions.
Coordinated Multidisciplinary Care
The center ensures rare disease patients receive comprehensive, individualized treatment plans through coordinated specialty care.
Services Offered:
Genetic counseling and diagnostic testing
Whole exome sequencing for diagnostic clarification
Clinical trial enrollment assistance
Case management and family support
Multidisciplinary specialty consultations
Research and Clinical Trials
The center supports hundreds of participants at any time, connecting Texas families to cutting-edge research opportunities. Research participation can provide access to investigational therapies; outcomes and timelines vary.
Contact Information:
Phone: 713-500-5764
Location: 6431 Fannin Street, Houston, TX
2. UT Southwestern Medical Center Rare Disease Programs
UT Southwestern treats the full spectrum of rare conditions through specialized clinics and multidisciplinary teams.
Specialty Clinics
Metabolic disorders clinic
Neuromuscular disease programs
Cancer predisposition syndromes
Skeletal dysplasias
Immune disorders
Medication Safety Programs
UT Southwestern provides comprehensive medication reviews, critical because some rare disease medications can interact with common drugs such as anticoagulants, anti-seizure therapies, or antibiotics. These interactions can be severe, requiring dose adjustment or substitution.
Best Practice: Full medication review and regular follow-up with a genetics or rare disease specialist are essential for patient safety.
3. Navigate Life Texas – Free Information and Resource Navigation
Navigate Life Texas provides free navigation services for Texas families, connecting them to resources, specialists, and financial assistance programs.
Services Provided
Information and referral services in English and Spanish
Connection to local support groups
School services navigation (IEP and 504 plan assistance)
Benefits and insurance guidance
Respite care program information
NORD Patient Assistance Program Access
Navigate Life Texas helps families apply for NORD assistance programs, which have provided long-standing support.
Coverage includes:
Medication costs
Insurance premiums
Travel expenses for clinical care
Copays and deductibles
Contact: Visit navigatelifetexas.org or call 2-1-1 Texas for referrals
4. Texas State Genetics Consortium – Comprehensive Statewide Network
The Texas State Genetics Consortium coordinates genetics services across Texas, recognizing that approximately 80% of rare diseases are genetic in origin.
Genetics Resources Directory
The TSGC maintains an updated directory of:
Genetic counselors throughout Texas
Genetic testing laboratories
Specialty genetics clinics
Research opportunities
Educational materials in English and Spanish
Clinical Trial Listings
The consortium connects families to ClinicalTrials.gov, listing actively recruiting studies. Families should maintain realistic expectations regarding diagnostic timelines and the availability of targeted therapies.
Access Resources: tsgc.org/genetics-resources-tx
5. NORD Financial Assistance Programs – Medication and Travel Support
The National Organization for Rare Disorders offers comprehensive financial assistance to reduce the burden of rare disease care costs.
Types of Assistance Available
Access to patient assistance programs can reduce financial and logistical barriers for rare disease families, allowing greater focus on treatment and daily living.
Programs include:
Life-saving medication coverage
Insurance premium assistance
Diagnostic testing support
Clinical trial travel reimbursement
Rare Caregiver Respite grants
Application Process
Patient assistance programs process applications based on individual program requirements. Start resource outreach upon diagnosis or suspected rare disease for optimal benefit timing.
Contact:
Phone: (800) 999-6673
Website: rarediseases.org/state-resource-center
6. Genetic Testing Access and Interpretation Services
Genetic testing provides diagnostic clarity after lengthy diagnostic journeys. About half of people living with rare diseases are children, making early genetic diagnosis critical for intervention planning.
Testing Options Available in Texas
Whole Exome Sequencing (WES):
Analyzes protein-coding regions of the genome
Identifies pathogenic variants in known disease genes
Covered by many insurance plans with prior authorization
Whole Genome Sequencing (WGS):
Most comprehensive genetic analysis available
Detects variants in non-coding regions
Research-grade sequencing available through university medical centers
HIPAA-Compliant Testing Protocols
All clinical genetic testing facilities follow strict HIPAA-compliant protocols to protect patient privacy. Genetic counselors help interpret test results and discuss implications for family members.
Insurance Coverage Considerations
Most commercial insurance and Medicaid cover diagnostic genetic testing when ordered by a physician with documented medical necessity. Prior authorization requirements vary by plan.
7. Experimental Therapies and Clinical Trial Navigation
For Texas families hearing "there are no treatment options," experimental therapies and clinical trials offer pathways forward. Nome's patient platform provides free AI-generated, expert-reviewed evaluations that map patient-specific experimental therapy options.
Understanding Clinical Trial Participation
Trial Types Available:
Gene therapy trials for single-gene disorders
Antisense oligonucleotide (ASO) development
Drug repurposing studies
Natural history studies
N-of-1 personalized trials
Finding Relevant Trials
Major research clinics support hundreds of participants simultaneously.
Search Resources:
ClinicalTrials.gov
Rare disease-specific registries
NORD Clinical Trial Finder
Medical center research coordinators
What Nome Offers Texas Families
When genetic mutations are identified, Nome analyzes whether personalized therapy is scientifically feasible and provides execution-ready next steps:
Free Summary Report: AI-generated assessment of custom therapy options, validated by PhD experts
Treatment Feasibility Scoring: Evidence-based evaluation of therapeutic approaches
Action Plan: Detailed roadmap including manufacturer contacts, regulatory pathways, and timelines
Ongoing Support: Live follow-up to address questions throughout the treatment development process
Nome coordinates geneticists, research labs, manufacturers, regulators, and providers—handling operational complexity that families cannot manage alone.
8. Personalized Medicine and N-of-1 Therapy Development
The science exists to develop patient-specific treatments, but traditional pharmaceutical companies lack financial incentive for ultra-rare conditions. Personalized therapeutics fill this gap.
Antisense Oligonucleotide (ASO) Therapies
ASOs are custom-designed genetic medicines that target specific mutations. In select cases, FDA has permitted individualized antisense oligonucleotide treatment via investigational new drug (IND) applications or Expanded Access.
Development Timeline:
Timelines vary widely by condition, safety studies, manufacturing, and regulatory review. In some documented N-of-1 cases, development proceeded in approximately one year, but many projects take longer.
Gene Therapy Options
Gene therapy replaces, inactivates, or introduces genes to treat disease. Multiple Texas medical centers conduct gene therapy trials for conditions including:
Spinal muscular atrophy
Duchenne muscular dystrophy
Inherited retinal diseases
Metabolic disorders
Hemophilia
When to Consider Personalized Approaches
Families should seek out multidisciplinary clinics and advocacy organizations to ensure that patients with rare diseases have the broadest possible support system available.
Consider personalized therapeutics when:
No FDA-approved treatments exist
Genetic mutation is well-characterized
Disease progression threatens significant morbidity
Family can access funding or grant support
Share your diagnosis with Nome to receive a free evaluation of whether personalized therapy could change your disease trajectory.
9. Texas Medicaid and CHIP Coverage for Rare Diseases
Texas Medicaid programs and the Children's Health Insurance Program (CHIP) provide coverage for eligible families facing rare disease expenses.
Medicaid STAR Programs
Texas operates managed care through regional STAR programs covering:
Doctor visits and specialist consultations
Hospital stays and surgeries
Prescription medications
Medical equipment and supplies
Home health services
CHIP Eligibility
CHIP covers children in families earning too much for Medicaid but unable to afford private insurance. Income limits vary by family size.
Prior Authorization and Appeals
Insurance denials are common for rare disease treatments. Texas families should:
Request detailed denial reasoning in writing
Obtain letters of medical necessity from specialists
Submit peer-reviewed literature supporting treatment
File appeals within specified timeframes
Request external review if internal appeals fail
Check your plan documents for exact deadlines; Texas also offers external review through the Texas Department of Insurance.
Documentation Requirements: Medical necessity documentation, treatment protocols, evidence of efficacy, and lack of alternative treatments strengthen appeals.
10. Educational Support Services for Texas Children
Texas Education Agency provides special education services for children whose rare diseases affect learning, mobility, vision, or hearing.
Individualized Education Programs (IEP)
IEPs provide legally binding educational accommodations for children with disabilities, including many rare diseases.
IEP Components:
Present levels of academic achievement
Annual goals and short-term objectives
Special education and related services
Accommodations and modifications
Participation in state assessments
504 Plans
Section 504 plans provide accommodations for students who don't require special education but need support due to disability.
Common 504 Accommodations:
Extended test time
Preferential seating
Modified assignments
Assistive technology
Medication administration
Services for Progressive Conditions
Children with degenerative conditions require ongoing IEP adjustments. Services may include:
Vision impairment specialists
Hearing loss support
Adaptive physical education
Occupational and physical therapy
Homebound instruction when necessary
Parent Rights: Texas parents have the right to request evaluations, participate in IEP meetings, and dispute decisions through mediation or due process hearings.
11. Travel and Lodging Assistance for Medical Care
Specialized rare disease care often requires travel to major medical centers. Multiple programs help Texas families manage these costs.
Ronald McDonald House Programs
Ronald McDonald Houses near Texas Medical Center in Houston and other major hospitals provide free or low-cost lodging for families.
Houston Ronald McDonald House:
Location: 1907 Holcombe Blvd, Houston, TX 77030
Capacity: Multiple locations serving Texas Medical Center
Eligibility: Child receiving treatment at nearby hospital
Cost: Sliding scale or free based on need
National Patient Travel Programs
Programs serving Texas families:
Corporate Angel Network (free flights on corporate jets)
Mercy Medical Airlift (commercial airline tickets)
National Patient Travel Center (coordinates multiple programs)
Gas card assistance programs through disease-specific foundations
Travel Reimbursement for Clinical Trials
Many clinical trials cover travel expenses for participants. Some NORD programs include travel assistance for clinical care, subject to program eligibility.
Application Process: Apply before travel when possible; keep detailed expense records including mileage, receipts, and travel dates.
12. Mental Health and Family Support Resources
The emotional toll of rare disease affects entire families. Texas offers specialized counseling and support services.
Rare Disease-Informed Counselors
Finding therapists who understand rare disease challenges improves treatment outcomes. Resources for locating informed providers include:
Psychology Today directory (filter by specialty)
Navigate Life Texas referrals
Disease-specific foundation recommendations
Hospital social work departments
Support Groups for Parents and Caregivers
Peer support reduces caregiver burnout and diagnosis grief. Texas Parent to Parent offers free peer support, training, and resource navigation for families through:
Regional support group meetings
Online communities
One-to-one parent matching
Sibling support programs
Addressing Caregiver Burnout
Continued collaboration between patient advocacy groups, clinical networks, and research funding agencies is vital to addressing treatment gaps and unmet needs in rare diseases.
Caregiver Self-Care Strategies:
Utilize respite care programs
Join caregiver support groups
Access mental health counseling
Set realistic expectations
Build support networks
NORD Rare Caregiver Respite Program provides grants to eligible families for temporary relief care.
Take Action Today
Texas rare disease resources span medical excellence, financial support, educational services, and innovative treatment development. You don't have to manage this journey alone.
Immediate Next Steps:
Contact McGovern Medical School or UT Southwestern for specialist referrals and coordinated care
Apply for NORD financial assistance and medication support programs
Connect with Navigate Life Texas for free information navigation and local resources
Request genetic testing if not yet completed—whole exome or genome sequencing provides diagnostic clarity
For experimental therapy exploration, share your diagnosis with Nome to see what personalized treatment options might be possible
One in ten Americans are affected by rare diseases, yet 95% have zero treatment options. The gap between science capability and treatment availability is closing through coordinated care, financial assistance, and personalized medicine approaches.
Your rare disease journey starts with clear information and actionable support. These Texas resources transform "no options" into next steps forward.
