Christine Falleti has spent much of her life combating the crippling effects of cystic fibrosis (CF). Now 34, she is painfully aware that she’s approaching the age when most people with CF die. Two friends with the disease already have. But last year she took part in a test of...
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A little girl who suffers from a serious lung disease and nearly died as a baby is on the path to a miracle recovery. Isabella Siddall, five, from Sutton, was born eight weeks before her due date, with underdeveloped lungs that led her to suffer from pulmonary hypertension (PT) and...
FOSTER CITY, Calif. & BEIJING – Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan). Maralixibat, an investigational, orally administered...
FOSTER CITY, Calif. — Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC). Mirum has also submitted an...
FOSTER CITY, Calif. — Mirum Pharmaceuticals, Inc., today announced top-line results of the Phase 2 EMBARK study evaluating LIVMARLI® (maralixibat) oral solution versus placebo given as an adjuvant therapy to Kasai surgery in patients with biliary atresia. The study did not meet its primary endpoint of mean change in bilirubin...
FOSTER CITY, Calif. — Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that the first participant has been enrolled in the BLOOM Phase 2 clinical study evaluating MRM-3379 in Fragile X syndrome (FXS). Caused by a mutation of the FMR1 gene, FXS is the most common inherited form...
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and...
FOSTER CITY, Calif. – Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare liver disease, today announced that it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (“PRV”) for $110 million. The PRV was granted by the U.S. Food and Drug Administration in...
FOSTER CITY, Calif. — Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from the Phase 3 MARCH-PFIC study evaluating LIVMARLI® (maralixibat) oral solution in patients with PFIC was published in The Lancet Gastroenterology and Hepatology. The MARCH-PFIC study was the largest, randomized, double-blind, placebo-controlled study in patients with PFIC,...
SOUTH SAN FRANCISCO, Calif. — Mission Bio, a leader in single-cell multi-omics solutions for precision medicine, announced a new publication led by Hervé Avet-Loiseau, MD, PhD, revealing new insights into disease progression of multiple myeloma (MM). Using Mission Bio’s single-cell DNA sequencing Tapestri® Platform, the study shows that solely targeting...