Every year on the last day of February, World Rare Disease Day is observed globally by over 100 countries, uniting people with rare diseases and their caregivers. This day raises awareness and encourages researchers, policymakers, healthcare professionals, and other leaders to address the needs of people living with rare diseases.

Rare diseases can be difficult to diagnose leaving many patients to go untreated for years. Most physicians lack education and training in how to identify, diagnose, and treat a rare disease because they encounter only a few in their careers. Patients also struggle to find educational resources that help them understand their diagnosis, treatment options and how to best communicate with their health care team.

PlatformQ Health’s work in rare disease education significantly increased after partnering with the National Organization for Rare Disorders. Together, over the years, NORD and PlatformQ Health have closely collaborated to produce a multitude of digital resources for healthcare professionals, patients, and families to facilitate timely diagnosis and improved care. Through this initiative and NORD’s close connections to other rare disease advocacy organizations and membership societies, we were able to expand our partnerships to create even more rare disease educational activities covering a dozens of different diseases and conditions in just the past year.

The initiative in rare disease continuing medical education is designed to help clinicians identify, diagnose, treat, and prescribe for these conditions. During many of these programs, clinicians can hear from patients and their families first-hand about their challenges and lived experiences, improving communication and strategies for shared decision-making. Comparably, PlatformQ Health’s rare disease patient and caregiver education aims to help them understand the disease they are facing and empower them to take control of their health.

We have found that accessible, digital, rare disease education can shorten the diagnostic odyssey and improve the overall quality-of-care patients receive.

Collaborating with NORD over the past year, together we created rare disease educational programs on the following diseases and conditions:

  • Dravet Syndrome
  • Spinal Muscular Atrophy (SMA)
  • Hemophilia A
  • Pediatric Neuroblastoma
  • von Willebrand Disease
  • Cold Agglutinin Disease
  • Peripheral T-Cell Lymphoma
  • Myelofibrosis
  • Sarcomas
  • Thyroid Cancer
  • NETS (neuroendocrine tumors)
  • GIST (gastrointestinal stromal tumors)
  • Bile Duct Cancer
  • Duchenne Muscular Dystrophy
  • Adult refractory epilepsy
  • Chronic inflammatory demyelinating polyneuropathy (CIDP)
  • Generalized Myasthenia Gravis
  • Progressive Myopathy

We are able to create impactful digital education programs on so many different rare diseases because of the support we receive from NORD and all of our other rare disease partners. These partners provide their expertise about each rare disease which helps us design education that is relevant and up to date with the needs of the community they serve and advocate for, and include: 

  • GBS|CIDP Foundation
  • NORD Rare Cancer Coalition
  • Cure SMA
  • Dravet Syndrome Foundation
  • Hemophilia Federation of America
  • National Hemophilia Foundation
  • Advances in Neuroblastoma Research Association
  • Parent Project Muscular Dystrophy
  • Solving Kids Cancer

In addition to providing impactful education, both NORD and the GBS|CIDP Foundation leveraged our proprietary engagement platform to host multi-day virtual conferences and widely attended webinars. Our platform gave the rare disease community a convenient, user-friendly way to connect and learn effectively, like they would have at an in-person event. PlatformQ Health is always eager for an opportunity to leverage our platform to strengthen our collaborations and support the rare disease community.

New Programs Coming Soon

Applying Enhanced Understanding of the Mechanisms of Disease in Generalized Myasthenia Gravis (gMG): Improved Diagnostics and Targeted Treatment Options

    • Produced in collaboration with NORD.

Progressive Myopathy: Could it be a Genetic Mitochondrial Disease? Registration link coming soon.

    • Produced in collaboration with NORD.

The 2021 Virtual Rare Cancer CongressComing Fall 2021 

Gene-Based Therapy 2021 SummitComing Fall 2021 

Rare Disease Digital Education On-Demand Library

Patient Education  in Rare Disease

Dravet Syndrome and Caregiving: Your Voices Heard and Answered

    • Produced in partnership with Dravet Syndrome Foundation and the National Organization for Rare Disorders.

Obtaining Support and Ongoing Care for Children with SMA after Gene Replacement Therapy: A 6 Module Series

    • Produced in partnership with Cure SMA and NORD.

Dravet Syndrome and Your Family: Diagnosis, Treatment, Family Needs, and Support: A 6 Module Series

    • Produced in partnership with the Dravet Syndrome Foundation and NORD.

A Closer Look at Hemophilia Management: Improving Outcomes with Personalized Care

    • Produced in partnership with the Hemophilia Federation of America, National Hemophilia Foundation and NORD.

Clinician Education in Rare Disease

Humanistic and Economic Burden of Dravet Syndrome: Multidisciplinary Approach to Recognize and Address

    • Produced in partnership with the Dravet Syndrome Foundation  and NORD.

Optimizing Prophylaxis Strategies of Hemophilia, A with or without Inhibitors: For Whom, When, and How Long?

    • Produced in partnership with NORD and the Hemophilia Federation of America.

Assessing the Impact of AEDs: What Constitutes Clinical Meaningful Effect for Dravet Syndrome

    • Produced in partnership with NORD and Dravet Syndrome Foundation.

Effective Administration and Management of Anti-GD2 Monoclonal Antibodies for Pediatric Neuroblastoma

    • Produced in collaboration with the Advances in Neuroblastoma Research Association.

von Willebrand Disease Management for Hematologists and Referring Clinicians: Diagnosis, Treatment, and Emerging Strategies

    • Produced in collaboration with NORD and the Hemophilia Federation of America .

Management of High-Risk Relapsed/Refractory Pediatric Neuroblastoma: Novel Agents and Patient Perspectives for Advanced Practitioners

    • Produced in collaboration with Solving Kids Cancer.

Spotlight on Rare Cancers: Advancing Novel Therapy and Patient Centered Management: Rare Blood Disorders, Leukemias, and Lymphomas

Cold Agglutinin Disease (CAD): The Role of the Complement Pathway in Treating a Rare Autoimmune Hemolytic Anemia

Addressing the Challenges of Peripheral T-Cell Lymphoma Management

Risk-Based Approach for Treatment of Intermediate Myelofibrosis

    • Produced in collaboration with the NORD Rare Cancer Coalition.

Spotlight on Rare Cancers: Advancing Novel Therapy and Patient Centered Management: Rare Solid Tumors

A Critical Update for the Multidisciplinary Team in Differentiating and Tailoring Management of Sarcomas

Where are we Headed in Thyroid Cancer? New and Emerging Treatment Approaches

All About NETs: Disease Pathway, Treatment, and Clinical Trials for GEP-NETs, Pheochromocytoma, and Paragangliomas

Reducing Diagnostic Challenges and Expanding Treatment Options for Advanced GIST

Molecular Profiling and Targeted Therapies Shifting the Paradigm for Cholangiocarcinoma (Bile Duct Cancer)

    • Produced in collaboration with the NORD Rare Cancer Coalition.

The Era of Disease-Modifying Therapy for Duchenne Muscular Dystrophy: Outlining Care Pathways for the Evolving Needs of the Patient

    • Produced in collaboration with NORD and Parent Project Muscular Dystrophy.

Managing Adult Refractory Epilepsy Patients: Why it is important to consider genetic epilepsy syndromes

    • Produced in collaboration with NORD.

Post-Gene Replacement Treatment SMA Phenotypes and Multidisciplinary Care

    • Produced in collaboration with NORD  and Cure SMA.

Understanding a Self-Infused CIDP Therapy Option

    • Produced in collaboration with the GBS|CIDP Foundation.

These rare disease focused educational programs are supported by Zogenix, Inc., Novartis Gene Therapies, Genentech, a member of the Roche Group, Takeda Pharmaceuticals U.S.A, Inc., Y-mAbs Therapeutics, Inc., Daiichi Sankyo, Inc.; Deciphera Pharmaceuticals, LLC.; Eisai Inc.; Epizyme Inc.; Innate Pharma, Inc.; Ipsen Biopharmaceuticals, Inc.; Karyopharm Therapeutics; Merck Sharp & Dohme, Corp., a subsidiary of Merck & Co., Inc.; Sanofi Genzyme; Seattle Genetics, Inc., PTC Therapeutics, Sarepta Therapeutics, AveXis, and CSL Behring.

Are you interested in collaborating or sponsoring with us to produce new rare disease education? Email us at digital@platformq.com.