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Rare Research Report
RDCRN
180 episodes
2 weeks ago
Rare Research Report features summaries of recent scientific publications from the Rare Diseases Clinical Research Network, which is funded by the National Institutes of Health. The network includes 20 active consortia—teams of researchers, patients, and clinicians—each focused on a group of rare disorders. Join us for new episodes each month.

Learn more about the RDCRN: https://www.rarediseasesnetwork.org
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All content for Rare Research Report is the property of RDCRN and is served directly from their servers with no modification, redirects, or rehosting. The podcast is not affiliated with or endorsed by Podjoint in any way.
Rare Research Report features summaries of recent scientific publications from the Rare Diseases Clinical Research Network, which is funded by the National Institutes of Health. The network includes 20 active consortia—teams of researchers, patients, and clinicians—each focused on a group of rare disorders. Join us for new episodes each month.

Learn more about the RDCRN: https://www.rarediseasesnetwork.org
Show more...
Science
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MGNet: Validating the Patient-Acceptable Symptom State (PASS) Question in Acetylcholine Receptor Myasthenia Gravis
Rare Research Report
1 minute
2 months ago
MGNet: Validating the Patient-Acceptable Symptom State (PASS) Question in Acetylcholine Receptor Myasthenia Gravis
New research from the Myasthenia Gravis Rare Disease Network (MGNet). This summary is based on a paper published in the European Journal of Neurology on June 25, 2025, titled "Validation of the 'Patient-Acceptable Symptom State' Question as Outcome Measure in AChR Myasthenia Gravis: A Multicentre, Prospective Study." 

Read the paper here. 

Learn more about MGNet. 

Transcript: 

New research from the Myasthenia Gravis Rare Disease Network (MGNet), a research group of the Rare Diseases Clinical Research Network.  Validating the Patient-Acceptable Symptom State (PASS) Question in Acetylcholine Receptor Myasthenia Gravis.

This summary is based on a paper published in the European Journal of Neurology on June 25, 2025.

Myasthenia gravis (MG) is a neuromuscular disorder caused by an autoimmune response which blocks or damages acetylcholine receptors in muscles, causing disabling weakness. New treatments have recently emerged for acetylcholine receptor (AChR) antibody-positive MG (AChR-MG). However, not all patients experience significant improvement, highlighting the importance of including the patient perspective in outcome evaluations.

In this study, researchers validated the Patient-Acceptable Symptom State (PASS) question as an outcome measure in AChR-MG. The team analyzed PASS responses among 173 patients with AChR-MG.

Results confirmed the PASS question as an effective, concise tool to assess AChR-MG patients' satisfaction with their disease control. Authors note that these findings also highlight the relevance of ocular complaints in patients' perception of MG burden.
Rare Research Report
Rare Research Report features summaries of recent scientific publications from the Rare Diseases Clinical Research Network, which is funded by the National Institutes of Health. The network includes 20 active consortia—teams of researchers, patients, and clinicians—each focused on a group of rare disorders. Join us for new episodes each month.

Learn more about the RDCRN: https://www.rarediseasesnetwork.org