Clinical Trial Paves the Way for a Groundbreaking New Myasthenia Gravis Treatment

A new drug for the rare neuromuscular condition has been approved by the FDA.

 
 

Biopharmaceutical company UCB recently shared important news that may affect those living with generalized myasthenia gravis (gMG): they’ve received approval from the U.S. Food and Drug Administration (FDA) for a groundbreaking treatment called ZILBRYSQ® (zilucoplan).

The FDA approved Zilucoplan based on data from a study called RAISE, published in The Lancet Neurology in May 2023. The study showed that Zilucoplan offered rapid and significant benefits to a wide range of gMG patients.

Know Rare is proud to have supported this development by recruiting patients for the RAISE Study. A key part of Know Rare’s mission is to help patients living with rare diseases to connect to clinical trials like this one that can have a positive impact on their well-being. If you’re interested in learning more about clinical trials and studies that might be a good fit for you, please contact us at support@knowrare.com.

MORE ABOUT ZILBRYSQ® (zilucoplan):

Zilucoplan is a self-administered once-daily therapy for adults with generalized myasthenia gravis that works by inhibiting complement-mediated damage to the neuromuscular junction, which is where your nerves and muscles communicate. This helps to prevent the damage and weakness caused by myasthenia gravis.  Unlike other treatments that need to be given intravenously, Zilucoplan is a subcutaneous (SC) peptide that you can inject under your skin. These developments mean that patients can take zilucoplan from their home, reducing the need for frequent hospital visits.

Know Rare is now recruiting for clinical trials related to these disease states:

  • Myositis (dermatomyositis, polymyositis, necrotizing myositis, myositis interstitial lung disease)

  • Myelin oligodendrocyte glycoprotein antibody disease (MOGAD)

  • Bullous pemphigoid (BP)

  • Tuberous sclerosis complex (TSC)

  • Becker muscular dystrophy (BMD)

If you’re interested in learning more, get in touch with Know Rare support here.

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