What a ‘Typical’ Day Is Like for My Son, Alfie, and Me
BEEP BEEP BEEP BEEP. My eyes spring open. It’s still dark. It’s not my alarm that I hear. “Wake up!” I say as I roll over and poke my husband. It’s time for him to go to work. This is how…
What a ‘Typical’ Day Is Like for My Son, Alfie, and Me
Second Phase 3 Study of Pamrevlumab Recruiting Boys With DMD
FibroGen has begun a second Phase 3 clinical trial of pamrevlumab, given with corticosteroids, among boys with Duchenne muscular dystrophy (DMD) who are able to walk. The trial, LELANTOS-2 (NCT04632940), is enrolling approximately 70 participants ages 6 to 12 at three U.S. sites — Arkansas…
#MDA2021 – Long-term Vamorolone Slowed Disease Progression in DMD Boys
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Long-term treatment with the experimental steroid therapy vamorolone safely and effectively delays motor function decline in boys with…
#MDA2021 – DMD Gene Therapy PF-06939926 Continues to Show Promise
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy PF-06939926 continues to demonstrate an acceptable safety profile and the potential for…
#MDA2021 – Gene Therapy SGT-001 Shows Continued Benefit in DMD Boys
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy SGT-001 continues to lead to improvements in physical functioning, disease-related biomarkers, and patient-reported outcomes…
#MDA2021 – Factors Likely to Predict Wheelchair Use With FSHD1 Detailed
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Factors related to age, medication use, and co-existing conditions predict the likelihood of a person with…
#MDA2021 – LGMD2E Gene Therapy SRP-9003 Shows Benefits After 2 Years
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. A potential gene therapy known as SRP-9003 led to sustained production of beta-sarcoglycan in muscle…
What Having Duchenne Muscular Dystrophy Feels Like
If you are familiar with the disease Duchenne muscular dystrophy, it’s easy to tell if someone has it. Enlarged calves, a waddling gait, the use of a wheelchair, and in some cases, a puffy face due to steroid use are all visual cues…
#MDA2021 – Plasma Exchange Enables Effective Gene Therapy Redosing
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Gene therapy redosing after therapeutic plasma exchange (TPE) — done to lower antibodies against…
#MDA2021 — Gene Therapy for DMD Exon Duplication Showing Promise in Trial
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Audentes Therapeutics’ experimental exon skipping gene therapy may safely and effectively increase dystrophin levels and stabilize muscle function…
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