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)

New article posted on Rare Disease Advisor: "Repeat Doppler Ultrasound Findings May Be Highly Variable." Read the full a...
26/09/2026

New article posted on Rare Disease Advisor: "Repeat Doppler Ultrasound Findings May Be Highly Variable." Read the full article here: https://buff.ly/LTWMqCc

Repeat fetal and maternal Doppler ultrasound readings, often performed in pregnancies at risk of HDFN, may vary considerably.

New article posted on Rare Disease Advisor: "Defining Muscle-Dominant AAV Using Systematic Muscle MRI." Read the full ar...
26/09/2026

New article posted on Rare Disease Advisor: "Defining Muscle-Dominant AAV Using Systematic Muscle MRI." Read the full article here: https://buff.ly/1FIaeYH

Systematic muscle MRI evaluations in patients with AAV should be performed rather than relying only on histopathologic biopsy or clinical suspicion.

New article posted on Rare Disease Advisor: "Intrauterine Transfusions Using ABO-Matched Rh-Negative Blood." Read the fu...
26/09/2026

New article posted on Rare Disease Advisor: "Intrauterine Transfusions Using ABO-Matched Rh-Negative Blood." Read the full article here: https://buff.ly/1tGqgMq

Intrauterine blood transfusions, used to treat conditions such as HDFN, may sometimes use the fetus’s ABO-compatible Rh-negative blood.

New article posted on Rare Disease Advisor: "Patients Report High Satisfaction With Subcutaneous ITP Treatment." Read th...
25/09/2026

New article posted on Rare Disease Advisor: "Patients Report High Satisfaction With Subcutaneous ITP Treatment." Read the full article here: https://buff.ly/mLawZ3g

Patients with ITP treated with a subcutaneous thrombopoietin receptor agonist (TPO-RA) report high treatment satisfaction and strong adherence.

New article posted on Rare Disease Advisor: "Combining MRI and Clinical Testing Helps Manage Dysphagia in Wilson Disease...
25/09/2026

New article posted on Rare Disease Advisor: "Combining MRI and Clinical Testing Helps Manage Dysphagia in Wilson Disease." Read the full article here: https://buff.ly/S9NTdij

Routine clinical test markers constitute the main early screening signal for swallowing difficulties caused by Wilson disease, research shows.

New article posted on Rare Disease Advisor: "Cough in PPF: Frequency, Causes, and Treatment." Read the full article here...
25/09/2026

New article posted on Rare Disease Advisor: "Cough in PPF: Frequency, Causes, and Treatment." Read the full article here: https://buff.ly/hPAkQmH

Cough is one of the most common and burdensome symptoms of PPF, affecting patients’ physical, emotional, and social well-being.

New article posted on Rare Disease Advisor: "Tofacitinib in RA-ILD: Trial Shows High Adverse Event Rates Alongside Bioma...
25/09/2026

New article posted on Rare Disease Advisor: "Tofacitinib in RA-ILD: Trial Shows High Adverse Event Rates Alongside Biomarker Reductions." Read the full article here: https://buff.ly/ORzVUuY

Patients with rheumatoid arthritis-associated ILD treated with tofacitinib had reduced disease activity and KL-6 levels, a study shows.

🔬 Created in partnership with the FDA and Critical Path Institute, NORD's free 3-part mobile-friendly course breaks down...
25/09/2026

🔬 Created in partnership with the FDA and Critical Path Institute, NORD's free 3-part mobile-friendly course breaks down the drug development process in English & Spanish!
- Foundations & Pre-Clinical Research
- Navigating Drug Review & Access
- The Basics of Clinical Trials
🔗 Enroll for free!https://buff.ly/goaHglv

-Path

How do rare disease therapies move from lab to market? In partnership with the U.S. FDA and Critical Path Institute, NOR...
25/09/2026

How do rare disease therapies move from lab to market?
In partnership with the U.S. FDA and Critical Path Institute, NORD offers a free, self-paced 3-part video series on drug development!

Available in both English and Spanish, this series covers pre-clinical research, review/access, and clinical trials.

Enroll for free today: https://buff.ly/goaHglv

-Path

New article posted on Rare Disease Advisor: "Ravulizumab and Eculizumab Reduce Steroid Use and MG-Related Hospitalizatio...
25/09/2026

New article posted on Rare Disease Advisor: "Ravulizumab and Eculizumab Reduce Steroid Use and MG-Related Hospitalizations: Real-World Data." Read the full article here: https://buff.ly/HifEwlP

Per US claims data, patients with MG who received ravulizumab or eculizumab saw reductions in steroid use, crises, and hospitalizations.

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