Next Step: Discover Annual Rare Disease Conferences and Research Updates

Staying connected to the latest research and the Hypophosphatasia (HPP) patient community can make a real difference in understanding your condition, finding support and shaping future treatments. Below you’ll find a guide to annual conferences, virtual events and research‐update resources geared toward HPP patients and families.

Why Attend Hypophosphatasia Patient Advocacy Conferences?

Participating in HPP–focused gatherings offers:

• Education: Hear experts present on enzyme replacement therapy, genetics and emerging treatments.
• Community: Meet fellow patients, caregivers and advocates who “get” the unique challenges of HPP.
• Empowerment: Learn advocacy skills—how to share your story with policymakers, researchers and funders.
• Clinical insights: Discover new trial opportunities and real‐world experiences from people living with HPP.
• Inspiration: See the progress made in other rare diseases and bring fresh ideas back to your local support network.

Key Annual Conferences for Hypophosphatasia Advocacy

1. Children’s Bone Health Foundation HPP Symposium

   • Focus: Deep dives into pediatric HPP, skeletal health and enzyme replacement updates.
   • Format: Half‐day patient/caregiver sessions followed by clinician‐only workshops.
   • Why go: Direct access to researchers who led asfotase alfa clinical trials.

2. American Society for Bone and Mineral Research (ASBMR) Annual Meeting

   • Focus: Broader bone diseases, but often features HPP poster and oral presentations.
   • Why go: Network with endocrinologists, geneticists and industry sponsors working on HPP therapies.

3. National Organization for Rare Disorders (NORD) Rare Diseases & Orphan Products Breakthrough Summit

   • Focus: Policy, drug development and patient‐powered research across all rare diseases.
   • Why go: Gain advocacy tools to accelerate HPP research and orphan drug designations.

4. Global Genes RARE Patient Advocacy Summit

   • Focus: Cross‐disease sessions on community building, mental health and digital tools.
   • Why go: Learn best practices from other rare disease advocates and adapt them for HPP.

5. International Congress on Rare Diseases & Orphan Drugs (ICORD)

   • Focus: Scientific advances in genetics, clinical trial design and global regulatory trends.
   • Why go: Understand where HPP fits into the larger orphan drug landscape and connect with global experts.

6. EURORDIS–Rare Diseases Europe Roundtables & Rare Disease Day Events

   • Focus: European policy initiatives and patient involvement in research planning.
   • Why go: If you’re based in Europe, influence Horizon Europe projects and steer funding toward HPP priorities.

Virtual and Online Events

Not everyone can travel to in‐person meetings. Virtual options include:

• HPP Foundation Webinars
  Quarterly online sessions covering bone health, dental issues and transition to adult care.

• NORD Virtual Summits
  Keynote recordings and live Q&A on FDA/EMA updates, trial enrollment and telehealth best practices.

• Orphanet Webinars
  Free seminars on genetic testing, natural history studies and patient registry development.

• RareDiseaseDay.org Digital Program
  Live‐streamed panels on mental health, financial planning and community activism for all rare diseases.

Staying Updated Between Conferences

Consistent updates help you spot new research and trial openings:

• Join Email Lists
  • HPP Foundation newsletter
  • NORD and Global Genes e-alerts
  • ASBMR member announcements

• Follow Social Media
  • Twitter/X hashtags: #Hypophosphatasia, #RareDiseaseDay
  • Facebook groups: HPP patient networks and parent support communities

• Monitor ClinicalTrials.gov and EU Clinical Trials Register
  Search “hypophosphatasia” to track enrolling studies of enzyme replacement, gene therapy and supportive care.

• Read Specialty Journals
  • Journal of Bone and Mineral Research
  • Orphanet Journal of Rare Diseases
  • Genetics in Medicine

• Listen to Patient‐Led Podcasts
  Firsthand stories often highlight real‐world challenges, treatment side effects and coping strategies.

Tips for First‐Time Conference Attendees

Preparing in advance ensures you get the most from each event:

• Review the Agenda Early
  Identify HPP sessions, poster presentations or workshops you can’t miss.

• Prepare Questions
  List specific inquiries about therapies, trial eligibility or multidisciplinary care teams.

• Connect Beforehand
  Use event apps or social media to arrange meet-ups with other HPP patients, researchers or advocates.

• Budget Strategically
  Apply for travel grants or patient scholarships—many organizations reserve funds for rare disease attendees.

• Bring Documentation
  A summary of your medical history or recent lab results can make one-on-one time with a specialist more productive.

Conducting a Symptom Review Online

If new symptoms arise or you simply want reassurance between doctor visits, you might consider doing a free, online symptom check, using the doctor approved Ubie Symptom Checker. It’s a quick way to gather insights before your next appointment.

Bringing It All Together

Attending Hypophosphatasia patient advocacy conferences and engaging with virtual events will keep you at the forefront of research, treatment options and policy efforts. By:

• Learning from global experts
• Connecting with an international community
• Sharpening your advocacy voice
• Tracking clinical trials and journal publications

—you can contribute to better care for yourself and others living with HPP.

Next Steps

1. Choose one or two upcoming conferences that match your needs.
2. Sign up for email updates from HPP and rare disease organizations.
3. Block time in your calendar for virtual webinars and workshops.
4. Prepare your questions and documents in advance.
5. Follow social media hashtags to stay informed in real time.

Finally, always speak to a doctor about any new, serious or life‐threatening symptoms. Your medical team can help interpret the latest research and decide how it applies to your unique health journey.