Jay Sandstrom

1 week ago | liverdiseasenews.com | Lindsey Shapiro |Margarida Maia |Jay Sandstrom |Marisa Wexler

The U.S. Food and Drug Administration (FDA) has approved an oral tablet formulation of Livmarli (maralixibat) to treat progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome. Livmarli, which has been available as an oral liquid formulation, is indicated in the U.S. for the treatment of itching, or pruritus, in PFIC patients 1 and older, and those with Alagille 3 months and older.

2 weeks ago | liverdiseasenews.com | Marisa Wexler |Lindsey Shapiro |Jay Sandstrom

A 34-year-old woman with Alagille syndrome and vascular problems — who had been implanted with a stent for her narrowed pulmonary arteries — experienced a blood clot that blocked her arteries after contracting COVID-19, a case study reports. A stent is a small, expandable metal mesh tube that can be inserted in narrowed or blocked blood vessels to keep them open. As with this patient, they can be used in the pulmonary arteries, which carry blood from the heart to the lungs.

3 weeks ago | liverdiseasenews.com | Lindsey Shapiro |Margarida Maia |Marisa Wexler |Jay Sandstrom

Livmarli (maralixibat) has been approved in Japan for the treatment of itching in people with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC), making it the first treatment available for these indications in the country. The medication will be commercialized by Takeda Pharmaceuticals under an exclusive licensing agreement with Livmarli’s developer Mirum Pharmaceuticals. Takeda submitted the application seeking Livmarli’s approval to Japanese regulators last summer.

1 month ago | liverdiseasenews.com | Jay Sandstrom |Lindsey Shapiro

I wish raising children would come with a checklist. Every day there are so many items to check off in addition to work and staying on top of the tasks around the house. When you add caring for a child with a rare genetic condition, that checklist becomes extensive. My son Finley was born four years ago with Alagille syndrome. He is my second child with my wife, Dani. When we decided to expand our family, we thought we had the parenting thing all figured out.

2 months ago | liverdiseasenews.com | Jay Sandstrom |Andrea Lobo |Margarida Maia

Tedious background music drones on repeat. Or worse, a repetitive voice recording reminds you that the lines are full and your call is important. For me, there aren’t many things worse than being put on hold during a call. Unfortunately, it comes with the territory when raising a child with Alagille syndrome. Our son Finley, 4, was hospitalized at 6 weeks old and then diagnosed with Alagille syndrome when he was 4 months old.