GM1 Gangliosidosis is a rare genetic condition that causes the body to retain fluids called interstitial fluid. This fluid serves to help with the movement of various elements throughout the body. However, if this interstitial fluid becomes excessive, it can cause fluid retention and swell in the limbs as well as around the heart. This can result in a condition known as GM1 gangliosidosis. Key companies in the field of healthcare and biopharmaceutical are focused on developing effective solutions for GM1 gangliosidosis. These efforts include continuous research & development activities, collaboration, and partnerships. Since there is a high prevalence of GM1 gangliosidosis, developing adequate therapeutics and drug discovery has become imperative. Recently, in December 2020, Sio Gene Therapies announced positive six-month follow-up data from a low-dose cohort of Phase I/II trial of its AXO-AAV-GM1 for GM1 gangliosidosis. Such factors can augment the growth of the GM1 gangliosidosis treatment market.
When it comes to regional footprint, North America is gaining rapid traction in the GM1 gangliosidosis treatment market. This is because of the high prevalence of GM1 gangliosidosis, particularly in the U.S. On contrary, Europe is exhibiting promising signs in the GM1 gangliosidosis treatment market due to the presence of robust healthcare infrastructure.
Recently, in November 2019, Axovant Gene Therapies Ltd. received the U.S. Food and Drug Administration (FDA)’s orphan drug designation for its investigational gene therapy AXO-AAV-GM1 for the treatment of GM1 gangliosidosis.
The most common treatment for GM1 Gangliosidosis is usually through medications. The only way to prevent GM1 Gangliosidosis from happening is by avoiding the triggers that can lead to its development. Some common things that can lead to this condition include being born with it, receiving diabetes, and having kidney problems. There is even a certain type of medication that can be addictive and lead to GM1 Gangliosidosis. However, there is low awareness regarding GM1 gangliosidosis, especially in emerging economies. This results in a low diagnosis rate and thereby even lower adoption of relevant treatment. Such factors can limit the growth of the GM1 gangliosidosis treatment market.
Since there is high demand for GM1 gangliosidosis treatment and medications, many clinical organizations are focused on inorganic strategies for the development of gene therapy. These strategies include collaborative agreements to develop an effective solution. Hence, such factors can accelerate the growth of the GM1 gangliosidosis treatment market shortly.