Rare Disease
Race to Save a Life: How an Act of Collaboration Led to a Bespoke Gene Therapy Treatment
Researchers know that a small act of generosity can have a big ripple effect.
Read MoreMass General’s Jay Iyer: Turning Personal Loss into a Mission to Transform PSP Care
Jay promised his grandfather he would find a cure for PSP, the rare neurological disease that took memories away from them.
Read MoreThese MGH investigators are at the forefront of rare disease research
At Massachusetts General Hospital, investigators are at the forefront of rare disease research. Here is what some of them said regarding their research, what motivates them and hope for a treatment or cure.
Read MoreWorld Medical Innovation Forum Outlines a Patient-Centric Vision for Gene and Cell Therapies
Researchers will need to identify new treatment strategies, work collaboratively to develop them into treatments for patients and ensure equity and access.
Read MoreSpotlight on Rare Disease Research at Massachusetts General Hospital
Our investigators are continuing to look for new insights into the causes of rare diseases and potential pathways to treatment.
Read MoreLooking for Insights into Mucolipidosis IV
Amanda Furness, PhD, is a postdoctoral research fellow in the Center for Genomic Medicine at Massachusetts General Hospital. Her poster, Therapeutic insight into Mucolipidosis IV via in vitro glia models, recently won an award at Mass General’s Research Fellow Poster Celebration. We asked Dr. Furness about her research: What problem(s) are you addressing with this…
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