Seelos Therapeutics Proposes New Treatment for Sanfilippo Syndrome

Seelos Therapeutics has won a meeting with the European Medicines Agency (EMA) to advance its therapy, trehalose, for the treatment of neuro-degenerative diseases, specifically Sanfilippo syndrome. At this meeting, which will happen in March, they hope to set up a clinical trial of their treatment in Europe. Along with this development in Europe, the biotechnology company also plans to expand the use of the treatment in America.

About Sanfilippo Syndrome

Sanfilippo syndrome, also known as mucopolysaccharidosis type III, is a disease in which the body cannot break down long chains of sugar molecules due to a lack of enzymes. These chains then accumulate, causing the symptoms of Sanfilippo syndrome. This syndrome comes in four forms: A, B, C, and D, with A being the most severe.

The onset of symptoms typically occurs about a year after birth, but they are most severe between the ages of two and six. Symptoms include:

  • Developmental issues
  • Behavioral and personality issues
  • Coarse facial features
  • Diarrhea
  • Sleep difficulties
  • Stiff joints that may not extend fully
  • Walking problems

This syndrome is inherited in an autosomal recessive pattern, meaning both parents must pass down the mutated gene in order for a child to be affected.

Doctors will diagnose Sanfilippo syndrome through a urinalysis, blood or skin sample, or genetic testing. Enzyme replacement therapy has been shown to be effective in other forms of mucopolysaccharidosis , but not this type. Treatment is symptomatic and supportive.

About Trehalose

Trehalose is an investigational molecule that has been indicated in the treatment of various neuro-degenerative diseases. It has been proven to reduce the amount of pathologic material as well as the clusters of misfolded proteins. It helps the body to get rid of damaged cells through the activation of transcription factor EB.

About the EMA Meeting

Seelos Therapeutics wants advice and help from the EMA to start a clinical trial of trehalose in Europe. They want to evaluate the effectiveness of this treatment on those who have types A and B of Sanfilippo syndrome. Not only will this meeting provide the biotechnology company with advice, but it will also give incentives to continue the development of the therapy. There is currently no approved treatment for this condition in Europe, making this meeting very important.

Trehalose in the U.S.

Trehalose is currently in the clinical trial stage in the United States, but Seelos hopes to expand this trial. They hope that they will soon be able to begin testing trehalose for types C and D of Sanfilippo syndrome and they also aim to include those with forms A and B who were unable to participate in the current trial.

Seelos Therapeutics aims to continue to develop this treatment for Sanfilippo syndrome, but it also has the potential to treat other conditions, such as Parkinson’s, ALS, Huntington’s disease, Alzheimer’s, oculopharyngeal muscular dystrophy (OPMD), spinocrebellar ataxia (SCA3), and Friedrich ataxia.

Find the original article here.


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