The Manton Center At Boston Children’s Hospital And Alexion Pharmaceuticals Partner In Artificial Intelligence Approaches To Rare Disease Diagnosis

February 27, 2018 BOSTON & NEW HAVEN, Conn.–(BUSINESS WIRE)–The Manton Center for Orphan Disease Research, the Innovation and Digital Health Accelerator at Boston Children’s Hospital, and Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced a collaboration to enable rapid and precise diagnoses

Vertex Announces Treatment with the NaV1.8 Inhibitor VX-150 Showed Significant Relief of Acute Pain in Phase 2 Study

-VX-150 was generally well tolerated and showed statistically significant relief of acute pain compared to placebo; study included an active reference arm of the opioid pain medicine hydrocodone+acetaminophen to support evaluation of VX-150 treatment effect- -Phase 2 study in acute

FDA Approves SYMDEKOTM (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene

– SYMDEKO is Vertex’s third medicine to treat the underlying cause of CF – – SYMDEKO to begin shipping to pharmacies this week – Feb 12, 2018 BOSTON–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA)

BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium™ 2018

Normalization of biomarker and liver size; stabilization of early cognitive effects suggested in preliminary data SAN RAFAEL, Calif., Feb. 7, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that it presented interim data from a Phase 1/2 trial for BMN 250,

Data Presented At WORLDSymposium™ 2018 Show Survival To 3 Years Of Age And Improvements In Liver Function In Infants With Lysosomal Acid Lipase Deficiency Treated With Kanuma® (Sebelipase Alfa)

February 6, 2018 NEW HAVEN, Conn.–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the combined interim data from two ongoing open-label studies, VITAL and CL-08, show a 3-year survival estimate of 68% in infants with rapidly progressive lysosomal acid lipase

BioMarin Announces 20 Presentations at 14th Annual WORLDSymposium™ 2018

Oral Presentations to Highlight Results from Studies for the Treatment of Sanfilippo Syndrome Type B, Morquio A Syndrome and CLN2 Disease, a Form of Batten Disease… SAN RAFAEL, Calif., Feb. 5, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that