CureDuchenne, a nonprofit advocacy group that supports and promotes research and better care and for people with Duchenne muscular dystrophy (DMD), has announced the launch of a three-year partnership with Baebies in which the company will add DMD to its expanded newborn screening service.
The goal is to make available an “easy-to-access” screening option for parents, CureDuchenne said in a press release.
“The ability to identify Duchenne-affected newborns within the first few weeks of life will empower parents to enroll their children in suitable clinical trials and start treatment as early as possible,” the release states. “It will also help the community to better understand the incidence of Duchenne and learn more about early symptoms of the disease.”
DMD is the most common form of muscular dystrophy and mainly affects young boys.
Early testing will also allow the community to benefit from the better understanding of the Duchenne’s symptoms at onset, and a “clearer understanding” of the disease’s phenotype (a patient’s observable characteristics) and genotype (genetic constitution.
Baebies is a growth-stage company that specializes in newborn screening and pediatric diagnostic tests.
“It is partnerships with organizations like CureDuchenne that will help us make the greatest possible impact,” said Richard West, co-founder and CEO of Baebies. “With a shared mission, this partnership will enable us to find these babies when they’re born and get them started on the treatment they need as soon as possible.”
CureDuchenne also announced plans for a series of partnerships and investments, such as this first undertaking with Baebies. To date, the nonprofit has leveraged more than $1.3 billion in follow-on investments to support research and other initiatives that further Duchenne MD treatment and care.
The group also plans to announce a fundraising and awareness-raising campaign.
“CureDuchenne’s business model is to accelerate programs, such as newborn screening, that will benefit the Duchenne community,” said Debra Miller, the founder and CEO of CureDuchenne. ” Newborn screening allows for early diagnosis which … can lead to better health outcomes for patients and avoidance of lengthy diagnostic odysseys, which delay crucial treatment.”
She added, “Our mission is to address the whole journey of a Duchenne patient, from community to physical therapy to clinical trials. Adding Duchenne newborn screening is a natural next step for us and we are excited about the benefits our partnership with Baebies will deliver to new parents and the Duchenne community.”
According to the Muscular Dystrophy Association, Duchenne and other muscular dystrophies are not among those on the national list of diseases recommended for screening in infants; only Pompe disease and spinal muscular atrophy are recommended.