Ophthalmology/Eye Diseases

Disease overviews and news and key expert videos focused on rare ophthalmology and eye disorders.
Apr 4, 2025| Posted in: Advocacy, Diagnosis, Ophthalmology/Eye Diseases

Outcomes of Intraocular Lens Exchange Surgery in Dead Bag Syndrome

A recent study evaluated outcomes of intraocular lens…
Apr 3, 2025| Posted in: Ophthalmology/Eye Diseases, Treatment

Botulinum Toxin Versus Strabismus Surgery for Acute Acquired Comitant Esotropia

Recent data was published detailing the results of…

Recent Videos

Optimizing Therapeutic Proteins Through PEGylation: Key Parameters and Impacts

The session will delve into the process of PEGylation, where polyethylene glycol (PEG) is conjugated to functional amino acid groups on the protein surface. This modification may enhance the properties of therapeutic proteins, offering advantages in stability, half-life, and immunogenicity.

Neuroblastoma

Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor in children. It is a neuroendocrine tumor that originates in neuroblasts or neural crest progenitor cells.

Optimizing the Efficacy and Safety of Therapy for Fabry Disease

Fabry disease is an inherited disorder that results from the buildup of a particular type of fat in the body’s cells, called globotriaosylceramide or GL-3. The disorder affects many parts of the body.

Epigenetic Modifiers as Therapeutic Targets

Gaucher disease (GD) is a genetic disorder in which glucocerebroside accumulates in cells and certain organs. The disorder is characterized by bruising, fatigue, anemia, low blood platelet count and enlargement of the liver and spleen, and is caused by a hereditary deficiency of the enzyme glucocerebrosidase, which acts on glucocerebroside.

FcRn and Myasthenia Gravis: Treatment Options

Richard J. Nowak, MD, MS, discusses the safety and efficacy of neonatal fragment crystallizable receptor (FcRn)-directed therapies for patient with myasthenia gravis.

Ophthalmology/Eye Diseases

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Safety and Efficacy of Zilucoplan to Treat Myasthenia Gravis: Results from the RAISE Trial

Improved Growth in Children Suffering from Fabry Disease Treated With Agalsidase Beta

Real-World Experience With C5 Complement Inhibition vs FcRn Antagonism in Myasthenia Gravis