Duchenne Muscular Dystrophy

2022 - 7 - 7

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Image courtesy of "PRNewswire"

Parent Project Muscular Dystrophy Announces Pediatric Certified ... (PRNewswire)

Leading Duchenne Organization Expands Certification of Clinics that Provide Optimal Care for People with Duchenne Muscular Dystrophy with 1st Clinic in St.

St. Louis Children's relies on generous donations to deliver specialized care for families in need, extend health care services to the community, and pursue research that promises to transform pediatric medicine. Schrader is thrilled to add St. Louis Children's to the growing network of Certified Duchenne Care Centers and to continue to expand the program to reach the large dystrophinopathy population currently under Dr. Zaidman's care. For more than 140 years, St. Louis Children's Hospital has provided exceptional care for children in all 50 states and 80 countries around the world. The center serves patients in need of both clinical care and those participating in research, as Children's is a large research hub in the region. In 2018, St. Louis Children's expanded with a state-of-the-art, 12-story medical tower. To learn more about PPMD's Certified Duchenne Care Center Program, visit PPMD's website. With its academic partner, Washington University School of Medicine, St. Louis Children's is consistently ranked among the nation's best pediatric hospitals by U.S. News & World Report. A member of BJC HealthCare, St. Louis Children's follows one simple mission – to do what's right for kids. Our advocacy efforts have secured hundreds of millions of dollars in funding and won five FDA approvals. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on YouTube. We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. "Being the only pediatric certified Duchenne care center in the St. Louis area is a tribute to the group of professionals who work at our clinic each day," said Zaidman. "We're honored to provide the highest level of care to our patients and families." WASHINGTON, July 7, 2022 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced the expansion of their renowned Certified Duchenne Care Center Program (CDCCP) with the certification of the clinic at St. Louis Children's Hospital. This is an exciting step for the CDCCP as it continues to expand its reach to include St. Louis and the surrounding region.

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Image courtesy of "Nature.com"

A medium-chain triglyceride containing ketogenic diet exacerbates ... (Nature.com)

Duchenne muscular dystrophy (DMD) is an X-linked recessive myopathy caused by dystrophin mutations. Although respiratory management has improved the ...

J. Gerontol. A Biol. Sci. Med. Sci. 74, 455–461 (2019). Sci Rep 12, 11580 (2022). https://doi.org/10.1038/s41598-022-15934-9 Cell 161, 1566–1575 (2015). The calorie ratios in MCTKD (KR 1.4) derived from fat, carbohydrate, and protein were 77%, 10%, and 13%, respectively (Supplemental Fig. 1B). The calorie ratios in ND (standardized AIN93M feeding) derived from fat, carbohydrate, and protein were 10%, 77%, and 13%, respectively. Lancet 353, 9–13 (1999). J. Nutr. Metab. 2018, 2630565 (2018). BMJ Open 8, e022572 (2018). Significant differences were observed in the strain rate variables; Radial strain rate S-peak (P = 0.048), longitudinal strain rate E-peak (P = 0.002), and transverse strain rate E-peak (P = 0.004). Furthermore, the MCTKD-fed DMD rats showed a significant decrease in LV fractional shortening (P = 0.011) in comparison to the ND-fed DMD rats (Fig. 2). Although the differences were not statistically significant due to the limited number of animals, most systolic and diastolic variables (Table 2) exhibited a trend toward worsening in the MCTKD-fed DMD rats compared to the ND-fed DMD rats (Fig. 3). Also, QRS duration in the MCTKD-fed rats was significantly longer than that in the ND-fed DMD rats (Fig. 1). All animal experiments were conducted in accordance with the guidelines for animal experiments at the National Institute of Advanced Industrial Science and Technology (AIST), The University of Tokyo, and international ARRIVE guideline 42. Histopathological analysis (Fig. 4A) revealed cardiomyocyte necrosis, infiltration of mononuclear cells, and fibrosis in the DMD rats, while they were not observed in the WT rats. In the comparison between ND-fed DMD rats and MCTKD-fed DMD rats, there were no significant differences in heart rate, P-wave amplitude, PR interval, or QT interval (Table 1). The R-wave amplitude in MCTKD-fed DMD rats was significantly lower than that in ND-fed DMD rats.

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Image courtesy of "Muscular Dystrophy News"

Reminding Myself That Not All Days With Duchenne MD Are Bad ... (Muscular Dystrophy News)

Bad days happen, columnist Betty Vertin writes. She describes one in particular after a son's anxiety attack, and how she survived it.

I managed to maneuver to the end of the conversation without telling her; it was not the time for my sensitive and sweet daughter to know a hard thing like that. I once wrote a blog post about it and promised to share it with her someday so that she knows how important she is to our family. It was a day that no book and no other parent could tell you how to navigate. I try to explain why, and they understand I need her to help and that she does it because she loves them. As a result, I was tired and grumpy for the remainder of the day. The experience took everything out of me, and I was running on fumes to begin with. Instead, I shared with her that she is special to us. When I am parenting alone while my husband is traveling, it’s a real challenge to balance Rowen’s and Callie’s needs and take care of the other three children still home. I was going on day six or seven as a single parent while my husband was traveling with one of my sons for baseball. That alone is grounds for me to feel tired and stressed. It was my last full day alone as my husband was due home early the next morning, and I thought we’d made it. My middle son with DMD, Rowen, who is 13 and nonambulatory, depends on me for a lot of help during the day, which is to be expected and is something I’m used to.

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