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New Therapy for Rare Spinal Muscular Atrophy Receives FDA Approval

The drug is the first to be approved to treat the rare genetic disease in both adults and children.

New Therapy for Rare Spinal Muscular Atrophy Receives FDA Approval

By: Sarah Hand, M.Sc.

Posted on: in News | Biotech News

According to researchers at King’s College London, almost three quarters of a person’s immune traits are governed by their genetic information. The study – which was published in the journal, Nature Communications – suggests that genes may play a bigger role in the development of the immune system than previously thought.

The researchers used data on 497 pairs of adult female twins collected through the TwinsUK cohort. In analyzing 23,000 immune traits, the researchers found that complex immune responses – known as adaptive immune traits – which develop after a person is exposed to a specific antigen, are strongly influenced by genetics.

“Our genetic analysis resulted in some unusual findings, where adaptive immune responses, which are far more complex in nature, appear to be more influenced by variations in the genome than we had previously thought,” said Dr. Massimo Mangino, lead researcher from King’s College London. “In contrast, variation in innate responses (the simple non-specific immune response) more often arose from environmental differences.”

The article also emphasizes the importance of environmental influences – such as diet, microbial exposure and infections – in childhood in developing the innate immune system. The findings could help inform future research in therapies for autoimmune diseases, including psoriasis and rheumatoid arthritis.

“Our results surprisingly showed how most immune responses are genetic, very personalized and finely tuned,” said Professor Tim Spector, Director of the TwinsUK Registry at King’s College London. “What this means is that we are likely to respond in a very individualized way to an infection such as a virus – or an allergen such as a house dust mite causing asthma. This may have big implications for future personalized therapy.”

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