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Printable Handouts
Navigable Slide Index
- Introduction
- Outline
- Myasthenia gravis (MG)
- Clinical presentation
- MG may mimic any eye movement abnormality
- Generalized weakness
- Diagnostic evaluation
- Pathophysiology of MG
- The site of pathology is the neuromuscular junction
- The neuromuscular junction
- Mechanisms of acetylcholine receptor antibody-mediated injury (1)
- Mechanisms of acetylcholine receptor antibody-mediated injury (2)
- Musk IgG4 antibodies
- Cellular mediators of MG
- Treatment of MG (1)
- Mortality of MG is low
- MG treatments and when they came into use
- Treatment of MG (2)
- Anticholinesterases
- Generalized MG corticosteroids
- The MGTX trial and MG treatment (1)
- The MGTX trial and MG treatment (2)
- Generalized MG immunosuppression
- Standard immunosuppressives
- Established acute therapies
- Recently FDA-approved
- Complement inhibition
- Complement system
- REGAIN study
- Eculizumab, FDA approved for use AChR-Ab
- Complement inhibition
- Most expensive US drugs
- Neonatal Fc receptor inhibitors
- Phase 3: multicenter, randomized, placebo-controlled of efgartigimod
- Better than placebo
- FcRN inhibition to be aware
- Economic analysis of eculizumab
- Comment
- B cell targeted therapy
- Randomized, blinded, placebo-controlled of rituximab for treatment resistant patients
- Phase 2: randomized, blinded, placebo-controlled of rituximab
- Efficacy and safety of rituximab for new-onset generalized AChR antibody positive MG
- Phase 3: randomized, blinded, placebo-controlled of rituximab
- Classification of evidences
- Conclusions
- Acknowledgements
Topics Covered
- Clinical presentation of myasthenia gravis
- Diagnosis of myasthenia gravis
- Pathophysiology of myasthenia gravis
- Standard and emerging therapies
- Clinical trials on the treatment of myasthenia gravis
Links
Series:
Categories:
Therapeutic Areas:
Talk Citation
Kaminski, H.J. (2023, August 31). Understanding myasthenia gravis and advances in its management [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved December 3, 2024, from https://doi.org/10.69645/EAKQ4638.Export Citation (RIS)
Publication History
Financial Disclosures
- Dr. Kaminski is principal investigator for the Rare Disease Network, MGNet supported by NIH grant U54NS115054 and a consultant for R43NS12432; is a consultant for Alnylam Pharmaceuticals, Roche, Takeda, argenix, Cabaletta Bio, UCB Pharmaceuticals, EMD Serono, Ono Pharmaceuticals, Gilde Healthcare and Admirix, Inc.
Other Talks in the Series: The Immune System - Key Concepts and Questions
Transcript
Please wait while the transcript is being prepared...
0:00
Hello. My name is
Henry Kaminski.
I'm a professor at George
Washington University,
and I've studied myasthenia gravis
for my entire medical career.
Today, I'll be talking to you about
understanding of myasthenia gravis
and recent advances
in its management.
0:19
A brief outline.
I'm going to discuss the
clinical presentation
and how myasthenia
gravis is diagnosed,
introduce the physiology
of myasthenia gravis,
and the standard of care
and emerging therapies,
which are very exciting
in our disease right now.
0:37
Let me first start with one of
the original descriptions of myasthenia
gravis by Sir Thomas Willis,
and allow me to read this.
"At this time I have under my
charge a prudent and honest woman,
who for many years
has been obnoxious to
this sort of spurious palsy.
Not only in her members,
but also in her tongue.
She for some time can speak
freely and readily enough,
but after she has spoke
long or hastily or eagerly,
she's not able to speak a word,
but becomes as mute as a fish.
Nor can she recover the use of
her voice under an hour or two."
This description has many key
features of myasthenia gravis.
The first one appreciates that
there are periods of normality.
She can speak
freely and readily,
but then she develops
fatigue to near muteness,
or entire muteness,
which I certainly have seen in
some patients during my career.
And then the weakness recovers.
A woman is described,
and so young women have a
propensity to develop this disease,
compared to men, in early years.
And Willis points out that
she's prudent and honest,
so there's a distinction
with psychiatric conditions.
Sadly, sometimes, myasthenia
gravis is confused with
and also delays the diagnosis
of actual myasthenia gravis.