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...
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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...
CAMBRIDGE, MA — When cancer patients undergo chemotherapy, the dose of most drugs is calculated based on the patient’s body surface area. This is estimated by plugging the patient’s height and weight into an equation, dating to 1916, that was formulated from data on just nine patients. This simplistic dosing...
CAMBRIDGE, MA — Patients undergoing chemotherapy often experience cognitive effects such as memory impairment and difficulty concentrating — a condition commonly known as “chemo brain.” MIT researchers have now shown that a noninvasive treatment that stimulates gamma frequency brain waves may hold promise for treating chemo brain. In a study...
Cambridge, Mass. – Patients with late-stage cancer often have to endure multiple rounds of different types of treatment, which can cause unwanted side effects and may not always help. In hopes of expanding the treatment options for those patients, MIT researchers have designed tiny particles that can be implanted at...
CAMBRIDGE, MA — For most patients, it’s unknown exactly what causes amyotrophic lateral sclerosis (ALS), a disease characterized by degeneration of motor neurons that impairs muscle control and eventually leads to death. Studies have identified certain genes that confer a higher risk of the disease, but scientists believe there are...
Cambridge, Mass. – A decade after scientists in The Picower Institute for Learning and Memory at MIT first began testing whether sensory stimulation of the brain’s 40Hz “gamma” frequency rhythms could treat Alzheimer’s disease in mice, a growing evidence base supporting the idea that it can improve brain health—in humans...