Tag Archive: rare and complex diseases

Nov 13 2017

Strengthening Duchenne Muscular Dystrophy Research: Antonio Rosato

From the time Antonio Rosato was diagnosed with Duchenne muscular dystrophy (DMD) at age 4½, his family was eager for him to participate in a clinical research trial. They wanted to give him access to the latest advances in pharmacological and disease management approaches for DMD, an opportunity that Antonio’s uncle Artie, who was diagnosed with the same neuromuscular disease four decades ago, did not have.

Read the rest of this entry >>

Nov 8 2017

Is Mindfulness Important for Mothers of Babies With Heart Conditions?

The Findings:

A cardiac intensive care unit (CICU) filled with tiny infants connected to collections of tubes and buzzing monitors can be an intimidating and overwhelming place for a mother who is worried about her newborn with congenital heart disease (CHD). A Children’s Hospital of Philadelphia team of nurse researchers conducted a qualitative study in which mothers of babies who have a complex heart condition described the post-diagnostic period, surgery, and the CICU stay as extremely stressful. The researchers also examined mothers’ coping mechanisms, and they identified mindfulness as a potential helpful early intervention tool to reduce mothers’ stress.

Read the rest of this entry >>

Oct 31 2017

A Day in the Life of a Clinical Research Coordinator

Editor’s Note: Our clinical research coordinators at Children’s Hospital of Philadelphia are a unique professional hybrid. Part researcher, healthcare professional, data coordinator, social worker, personal coach, and travel concierge, they go above and beyond their job descriptions in order to help families navigate the complexities of participating in clinical research studies. We invited Joshua Zigmont, RN, BSN, who has been a nurse research coordinator for seven years, to describe a typical day on the job. The first thing he told us is that the most consistent thing about the position is its inconsistency: “You have to be flexible because you never know when a curveball is coming,” Zigmont said. So while no day is ever typical, here is a glimpse of a day in the life of a clinical research coordinator.

Read the rest of this entry >>

Oct 27 2017

Friedreich’s Ataxia Community Stays on Top of Science and Stays in Touch

Patients and families dealing with the rare, debilitating disease Friedreich’s Ataxia showed their true strength at the 10th Annual Friedreich’s Ataxia Symposium held in King of Prussia, Pa. The FA community is a steadfast supporter of research, and they have been gathering for a decade to spend a day with each other building friendships and learning the most up-to-date information on the therapeutic approaches and scientific studies being conducted in the field of FA.

Read the rest of this entry >>

Jan 3 2017

Looking Ahead to Having a Bigger Impact and Greater Success

I always look toward the new year with great optimism and hope, to the promise and potential it brings, and to the experiences that will shape us individually, professionally, and organizationally.

Read the rest of this entry >>