1. Healthcare burden among individuals with Angelman syndrome: Findings from the Angelman Syndrome Natural History Study. Issue 7 (14th May 2019) Authors: Khan, Nasreen; Cabo, Raquel; Tan, Wen‐Hann; Tayag, Regina; Bird, Lynne M. Journal: Molecular genetics & genomic medicine Issue: Volume 7:Issue 7(2019) Page Start: n/a Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Outcomes after 18 months of eliglustat therapy in treatment‐naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial. Issue 11 (3rd October 2017) Authors: Mistry, Pramod K.; Lukina, Elena; Ben Turkia, Hadhami; Shankar, Suma P.; Baris, Hagit; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Pastores, Gregory; Petakov, Milan; Assouline, Sarit; Balwani, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Gaemers, Sebastiaan J. M.; Tayag, Regina; P... Journal: American journal of hematology Issue: Volume 92:Issue 11(2017:Nov.) Page Start: 1170 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗