by Peter Ciszewski | Feb 6, 2020
Maria Picone is the mother of a child with Prader-Willi syndrome as well as founder/CEO of TREND Community – a company that collects anecdotal information from patients and caregivers so that the information can be quantified and assessed more efficiently. The...
by Peter Ciszewski | Feb 5, 2020
Dianne Linnehan, Director of Operations of the Phelan-McDermid Syndrome Foundation is also the parent of a person with Phelan-McDermid syndrome, and as she explains in this interview, her daughter provides her with immeasurable strength and joy, despite the...
by Peter Ciszewski | Feb 4, 2020
Andra Stratton, president of Lipodystrophy United provides an overview of lipodystrophy. Lipodystrophy is a rare medical condition in which persons have an abnormal distribution of fat in the body. That abnormality can present itself in many ways. People with...
by Peter Ciszewski | Feb 3, 2020
Sandy Macrae, PhD, Chief Executive Officer of Sangamo Therapeutics, a company developing genetic therapies for a variety of rare conditions, including hemophilia A, Fabry disease, beta-thalassemia, sickle cell disease, mucopolysaccharidosis I (MPS I) and MPS...
by Peter Ciszewski | Jan 31, 2020
Snejana Krassova, MD of Bayer Pharmaceuticals talks about the reality that some people have, or can develop, antibodies to the vectors that are commonly used for gene therapy. Bayer is currently developing a gene therapy for hemophilia A, and as Dr. Krassova...
