The Lysosomal Disease Network Awards LDN Fellow for 2017-2018

The Lysosomal Disease Network (LDN) is pleased to announce that it has selected Laura Adang, MD, PhD of The Children’s Hospital of Philadelphia for a fellowship that provides $50,000 for lysosomal disease clinical research. Her research project is entitled “Metachromatic leukodystrophy: characterization of genetic mutations, age of onset, and clinical subtypes.” Dr. Adang’s mentor for this project is Dr. Adeline Vanderver, also with The Children’s Hospital of Philadelphia. Dr. Adang’s fellowship period is August 1, 2017 – July 31, 2018. Click here for more information about the LDN’s fellowship opportunities.

The project is a multi-center retrospective natural history study to characterize the disease course in patients affected by metachromatic leukodystrophy (MLD). It will use statistical modeling to analyze collected clinical data to classify distinct subpopulations within the heterogeneous MLD population. Ultimately, it will evaluate the correlation and distinctions between the subpopulations with respect to genetic, radiographic, and ancillary clinical phenotypes, including gallbladder and renal involvement. Dr. Adang said the project goals include “validating or possibly redefining the clinical subtypes of metachromatic leukodystrophy. This can be used to design future studies and therapeutic trials. Importantly, with a better understanding of metachromatic leukodystrophy, we will be able to offer our families better anticipatory guidance, establish appropriate standards of care, and design better future therapeutic trials.”

Dr. Marc Patterson, Director, Education Core of the Lysosomal Disease Network, said “The Lysosomal Disease Network looks forward to a very productive research project, and wishes Dr. Adang the greatest success in achieving the goals of this research project, with a peer-reviewed publication summarizing the findings. Dr. Adang will also participate in the NIH-funded RDCRN Rare Disease Clinical Research Training Program, which is also open to new clinical investigators.”

LDN Fellow Named as Outstanding Mentor

Reena Kartha, PhD, a former LDN Fellow, is a Research Assistant Professor with the University of Minnesota College of Pharmacy’s Department of Experimental and Clinical Pharmacology, and a Principal Scientist for the College of Pharmacy’s Center for Orphan Drug Research. Her research focuses on the role of oxidative stress and inflammation in neurodegenerative conditions. Dr. Kartha also generously helps interview applicants for the University of Minnesota Center for Clinical and Translational Science Institute’s summer programs. Dr. Kartha has been named the 2017 CTSI Outstanding Junior Mentor.

Dr. Kartha receives her award for Outstanding 2017 Junior Mentor

Reena Kartha, PhD receives the Univ. of Minnesota’s CTSI 2017 Outstanding Junior Mentor award. Reena Kartha, PhD (right), and Anne Marie Weber-Main, co-director of CTSI’s mentoring program (left).

Comments from nominating mentees included: “Dr. Kartha is one of the most intelligent women I have ever had the honor of meeting and I am inspired by her internal strength, wisdom, confidence, and grace. She is someone I hope and aspire to become, and not just from a researcher perspective, but as a person.”


“What distinguishes Dr. Kartha from most other mentors is the relationships she develops with her mentees. The net result is a string of trainees who have grown substantially as researchers and, more importantly, and have developed a sense of curiosity and excitement about clinical science.”

The Lysosomal Disease Network congratulates Dr. Kartha, and is proud to have chosen her as one of its 2016-2017 Fellows. Dr. Kartha also is a graduate of the RDCRN’s Rare Disease Clinical Research Training Program. She will be presenting her research at the NORD Rare Diseases and Orphan Products Breakthrough Summit, October 16-17, 2017 in Washington, D.C.