Azafaros, a clinical-stage biotechnology company developing novel therapies for rare lysosomal storage disorders (LSDs), today announced that it will present at the 2025 BIO International Convention ...

GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases ... including gangliosides in the CNS. The onset of disease can happen throughout the lifespan of an affected individual ...

“Fact Checking” with the Patient Community: An Industry-Advocacy Collaboration for Optimizing Trial Design: This poster is the result of a collaboration between Azafaros and two US organisations, ...

In a pioneering study, researchers at McMaster University have discovered a possible treatment for Sandhoff and Tay-Sachs ... late-onset patients provided crucial insights into how the disease ...

Researchers have identified a potential treatment for Sandhoff and Tay-Sachs diseases ... cord neurons in late-onset patients provided crucial insights into how the disease progresses throughout ...

In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ... onset patients provided crucial insights into how the disease ...

Most children do not survive past age 5. For adults afflicted by late onset Tay-Sachs, symptoms are often confused with mental illness or other diagnoses. Tay-Sachs disease creates an enzyme ...

An even rarer type of Tay-Sachs is called “late onset” or “adult onset.” It can be hard to diagnose. Like the version of the disease that affects infants, Tay-Sachs that starts later in ...

Late-onset Tay-Sachs is the least severe form of the disease. Symptoms usually begin to appear in the 30s. Until then, the body has been adequately producing lysosomes. As a result, symptoms of ...

Late-onset Tay-Sachs disease (LOTS) is extremely rare ... Case Summaries We present four adult patients with known LOTS from four unrelated non-consanguineous families. Each had neurophysiological ...

Anna Pak Poy's son Sebby was diagnosed with an ultra-rare disease called Tay-Sachs when he was just seven ... before their symptom onset has progressed too far." Peppa Rasmussen-Breitinger ...

Late onset Tay-Sachs disease is a neurodegenerative condition that runs in families. This form of Tay-Sachs disease progresses slower than other forms and doesn’t typically shorten your life ...