Jul 26, 2020 | NEWS
Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trial. Please click here to read.
Oct 8, 2018 | NEWS
Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate Data from IND-enabling studies in three species support moving SPK-3006 into the clinic in 2019 PHILADELPHIA, Oct. 08, 2018—Spark Therapeutics, a fully integrated, commercial...
Sep 10, 2018 | NEWS
CRANBURY, N.J., Sept. 10, 2018—Amicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, announced today regulatory and clinical advancements in its development program AT-GAA for...
Jun 26, 2018 | NEWS
Researchers from the Erasmus MC in Rotterdam, Netherlands, have succeeded in generating muscle cells from skin cells. This is significant as skin cells can be relatively easily obtained from a skin biopsy. The team developed a protocol to obtain pure muscle cells (ie...
Jan 11, 2016 | NEWS
Reveglucosidase Alfa Trends Positive in Respiratory Muscle Strength and Endurance Endpoints in Patients Previously Treated With Enzyme Replacement Therapy SAN RAFAEL, Calif., Jan. 11, 2016 (GLOBE NEWSWIRE)—BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced...