This April, we rolled up our sleeves, got a little crafty and gave back with heart in recognition of National Volunteer Month. Most recently, employees at our San Rafael campus teamed up with California nonprofit Peninsula Bridge to assemble hands-on STEM kits for elementary school students in underserved communities. The kits included everything students need to launch balloon rockets, put together squishy circuits, build catapults and experience how fun engineering can be.

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Antisense oligonucleotides are small genetic sequences with big potential. By targeting specific sequences of messenger RNA (mRNA), they can fine-tune genetic instructions, helping regulate production of a specific protein in a targeted way. One powerful application is exon skipping – allowing cells to bypass specific genetic sequences and produce a more functional protein. By unleashing the potential of genetic science, our researchers are pushing the boundaries of what’s possible in treating genetically defined conditions.

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Congratulations to our friend Kendra Gottsleben on another successful and inspiring edition of “The Style Show: A Runway to Empower” this past weekend. Kendra started this event, held annually in her hometown of Sioux Falls, South Dakota, three years ago to promote inclusion and accessibility in fashion. In front of a sellout crowd of 250, this year’s show featured 32 models of various backgrounds and abilities, including people with rare genetic conditions like Kendra, who is living with MPS VI.

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Meet Kenneth: a dog lover, gamer, 1970s movie aficionado and record collector, who lives his life to the fullest while managing hemophilia A. As Kenneth reflects on his experience growing up and how living with hemophilia has evolved, he is grateful for the global online community that now exists.

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