Rare Disease Advisor

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Rare Disease Advisor Trusted knowledge base of resources & information, focused on treating & diagnosing .

Rare Disease Advisor is an online resource that provides health care professionals with comprehensive information on important principles of care in rare diseases as well as up-to-date clinical news on the diagnosis and treatment of these conditions. Website features include:

• Daily news
• Feature articles on key issues in rare disease
• Live conference coverage
• Expert opinion articles, including exclusive guest editorials from leading rare disease experts
• Concise drug monographs
• Medical slideshows
• Videos
• Continuing medical education (CME) activities
• And more...


Coverage of the following Rare Diseases:

Cardiology:
Long Chain Fatty Acid Oxidation Disorder (LCFAOD)
Pompe Disease

Gastroenterology:
Gastrointestinal Stromal Tumor (GIST)

Hematology:
Hemophilia
Sickle Cell Disease (SCD)
Systemic Mastocytosis (SM)

Hepatology:
Alagille Syndrome (ALGS)
Lysosomal Acid Lipase Deficiency (LAL-D)

Immunology:
Cold Agglutinin Disease (CAD or CAgD)
Hereditary Angioedema (HAE)
Myasthenia Gravis (MG)

Neurology:
Duchenne Muscular Dystrophy (DMD)
Multiple Sclerosis (MS)
Spinal Muscular Atrophy (SMA)
Hereditary Transthyretin Amyloidosis (hATTR)
Neuromyelitis Optica Spectrum Disorder (NMOSD)

Oncology:
Cholangiocarcinoma (CCA)
Medullary Thyroid Carcinoma (MTC)
Diffuse Large B-Cell Lymphoma (DLBCL)

Pulmonology:
Alpha-1 Antitrypsin Deficiency (AATD)
Cystic Fibrosis (CF)
Idiopathic Pulmonary Fibrosis (IPF)
Pulmonary Arterial Hypertension (PAH)

🎉 "Ten years ago, RDDS started as an experiment in bringing advocates and industry into the same room. Today, it's a pro...
21/08/2026

🎉 "Ten years ago, RDDS started as an experiment in bringing advocates and industry into the same room. Today, it's a proven launchpad for patient-led and patient-partnered research, said Charlene
Son Rigby, CEO of Global Genes. Rare disease progress depends on advocates who know how to navigate research, build the right partnerships, and drive development forward. Ten years in, that's the
commitment RDDS continues to deliver on."
Join RDDS' Fireside Chat with Charlene Son Rigby and Dr. Tim Yu, Boston's Children Hospital on Sept. 10th 2026:
https://buff.ly/ymUoJkK


www.globalgenes.org

💡 You are not alone! These RARE facts show the impact of rare disease. Global Genes shares these statistics with the ra...
17/08/2026

💡 You are not alone! These RARE facts show the impact of rare disease. Global Genes shares these statistics with the rare disease community to let them know that RARE really is EVERYWHERE. Visit the Global Genes website for more facts and resources:
https://buff.ly/rJXLdof


www.globalgenes.org

🎊 This month’s Rare Advocacy Spotlight honors Global Genes! For two decades they’ve eased the burden for people with rar...
14/08/2026

🎊 This month’s Rare Advocacy Spotlight honors Global Genes! For two decades they’ve eased the burden for people with rare diseases worldwide—supporting 400M+ individuals with education, training, and resources that build community and drive research. 💫 Check out our 10th RAS feature: https://buff.ly/dL013U0

www.globalgenes.org

Finding the right care is critical. The wAIHA Warriors Hematologist Directory is compiled from patient referrals, medica...
31/07/2026

Finding the right care is critical. The wAIHA Warriors Hematologist Directory is compiled from patient referrals, medical journal publications, and clinical trial participation. Connect with wAIHA expert care today!

https://buff.ly/ilT18jM

Advocacy is a core pillar of wAIHA Warriors. The organization looks ahead to "Warriors on the Hill" Sept 16–18, 2026 — c...
27/07/2026

Advocacy is a core pillar of wAIHA Warriors. The organization looks ahead to "Warriors on the Hill" Sept 16–18, 2026 — continuing to amplify patient voices and drive inclusive evidence and meaningful, informed care for our community. Mark your calendar to join in-person!

https://buff.ly/OZLGwQE

The 2026 wAIHA Warriors' 4th Annual Patient Meeting brought together patients, care partners, and experts to explore war...
24/07/2026

The 2026 wAIHA Warriors' 4th Annual Patient Meeting brought together patients, care partners, and experts to explore warm autoimmune hemolytic anemia. Missed it? Watch the recording to learn about symptoms, current research, treatment options, and patient stories:

https://buff.ly/eVGxTSH-

💡 Recognizing wAIHA is the first step on a patient's path. The wAIHA Warriors guides those newly diagnosed through sympt...
17/07/2026

💡 Recognizing wAIHA is the first step on a patient's path. The wAIHA Warriors guides those newly diagnosed through symptom care and treatment choices — a concise resource with key facts on who it affects, common signs, and typical therapies.

https://waihawarriors.org/what-is-waiha%3F
#

🌟 This Month’s Rare Advocacy Spotlight: The wAIHA Warriors 💫 We are proud to shine a light on the wAIHA Warriors who ens...
16/07/2026

🌟 This Month’s Rare Advocacy Spotlight: The wAIHA Warriors

💫 We are proud to shine a light on the wAIHA Warriors who ensure that no one affected by warm Autoimmune Hemolytic Anemia has to face this disease alone. Join us this month as we highlight their dedication to education, support, and advocacy.

Read the story here: https://buff.ly/XHsPWiw

⏳Have you taken the Parents Speak Survey II? Your perspective is vital for shaping future patient care and research—shar...
30/06/2026

⏳Have you taken the Parents Speak Survey II? Your perspective is vital for shaping future patient care and research—share your voice before it closes. DDE's leadership and science team are counting on you!
https://buff.ly/W1HSWX2

, PhD, , PhD, Wojnaroski, PhD, , PhD, , PhD, , MS, , MPH, , PhD, , PhD, , PhD

Rare Advocacy Spotlight highlights 13-year-old Elliott Conecker alongside his devoted sister Vega. Diagnosed with SCN8A ...
26/06/2026

Rare Advocacy Spotlight highlights 13-year-old Elliott Conecker alongside his devoted sister Vega. Diagnosed with SCN8A before his first birthday -- back when only 12 cases were documented -- Elliott lives with one of the most severe SCN8A mutations, including vision impairments and developmental delays that limit his ability to connect with family and peers.
Meet Elliott and his devoted family: https://buff.ly/ZFnPEkY

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