As someone being treated for a rare disease in Minnesota, I am experienced with prescription drug costs. Living with cystic fibrosis for decades now, its related pain, treatments and challenges, I can appreciate the need to lower costs for medications. The question is, what is the best way to do...
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Philadelphia — Every cause needs a champion. That’s why Minnesota Twins outfielder Michael A. Taylor has launched a season-long home run challenge to support Uplifting Athletes and the rare disease community. Inspired by his sister Danielle, Taylor will use his platform as a Major League Baseball player to inspire hope...
CAMBRIDGE, Mass. & HAIFA, Israel – Minovia Therapeutics, a clinical-stage company focused on the development of cell-based Mitochondrial Augmentation Therapy (MAT) to treat mitochondrial diseases, announced today the appointment of a key member of Management for the Company’s next phase of growth. In anticipation of feedback from the FDA on...
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...
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...
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...