Rare Disease Day Partner
Raise Your Hand for Rare Disease Research! Duke Research Leads to "Extraordinary Measures" Film LysoSENS: Destroying junk inside cells. Lysosomal Activity Declines With Aging Harrison Ford, Brendan Fraser in Pompe disease movie Adult Stem Cell Research Gives Heart Disease Patient a Miracle GM-gangliosidosis in Alaskan Huskies: Clinical and Pathologic Findings BioMarin's Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA Calling all Families Affected by ASMD Niemann-Pick Disease (Types A and B) - Can you Help? (.pdf)
Raise Your Hand for Rare Disease Research! Duke Research Leads to "Extraordinary Measures" Film LysoSENS: Destroying junk inside cells. Lysosomal Activity Declines With Aging Harrison Ford, Brendan Fraser in Pompe disease movie Adult Stem Cell Research Gives Heart Disease Patient a Miracle GM-gangliosidosis in Alaskan Huskies: Clinical and Pathologic Findings BioMarin's Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA Calling all Families Affected by ASMD Niemann-Pick Disease (Types A and B) - Can you Help? (.pdf)