At the one year mark, Sarepta Therapeutics’ SRP-9001 micro-dystrophin gene therapy continues to show positive results in four boys with Duchenne muscular dystrophy (DMD) enrolled in a Phase 1/2 study. The therapy improved the boys’ motor abilities and…
People with neuromuscular disorders such as muscular dystrophy are not at a higher risk of acquiring COVID-19, but they do face greater challenges should they become infected. Creating tailored plans to manage risk and adhering to them is especially important. This is the broad take-home message of a…
My parents tried to shield me as long as they could from data that revealed the average life expectancy of patients with Duchenne muscular dystrophy (DMD). Once I grew older and more curious, I finally came across those numbers, and they scared me. I…
The European Commission has granted orphan drug designation to viltolarsen, a treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, Nippon Shinyaku announced in a press release. The designation is given to medicinal compounds meant to treat rare conditions that are life-threatening or…
On May 1, I became eligible for Medicare. This is one of those milestones in life. I was able to access this benefit a few years early because of my muscular dystrophy. I am now on Social Security Disability Insurance (SSDI). The way it works is that after…
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.
A single administration of the gene therapy SRP-9003 at a low dose continues to lead to significant improvements in functional measures in three children with limb girdle muscular dystrophy (LGMD) type 2E, one-year data from a Phase 1/2 trial show. When administered at a high dose in three…
Positivity is a characteristic that I naturally possess. I seldom struggle to find the bright side of situations. Although this attribute has saved me innumerous times from acquiring a negative disposition, I have not been completely unscathed by depression and anxiety. My mental illness began early…
Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University related to the development of potential gene therapies for LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). Included are a license agreement as well as a collaboration with Peter Yurchenco, MD, PhD, a professor at Rutgers’ Robert Wood Johnson Medical…
Parent Project Muscular Dystrophy (PPMD) and Duchenne UK have joined forces to offer up to $1 million in funding for gene therapy research in Duchenne muscular dystrophy (DMD). The organizations are specifically looking for proposals that address the immune system’s response to gene therapy, a major challenge…
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