Muscular Dystrophy

Although a small sample of 5 patients with DMD, results showed significant expression of microdystrophin and reductions in creatine kinase, a biomarker of muscular distress, in GTN0004-treated patients.

Deflazacort, a corticosteroid approved for children with Duchenne muscular dystrophy, has been on the market since 2017 under the name Emflaza.

The FIREFISH study showed that most children maintained or improved motor functions and feeding abilities over five years.

Following the mixed results, NS Pharma is working with the FDA to determine how to proceed with viltolarsen.

Ataluren, which was conditionally approved in Europe in 2014, had its real-world use backed by data from the STRIDE registry.

Dyne aims to pursue expedited approval pathways for DYNE-251, leveraging dystrophin as a surrogate biomarker.

DYNE-101 demonstrated dose-dependent splicing correction, with the 5.4 mg/kg cohort showing a 27% mean correction at 3 months.

The phase 3 REACH trial investigating Fulcrum Therapeutics’ losmapimod in patients with facioscapulohumeral muscular dystrophy remains on track with topline data anticipated in the fourth quarter of 2024.

Avidity Biosciences anticipates to provide new cohort data from the phase 1/2 EXPLORE44 trial assessing AOC 1044 in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the second half of 2024.

Solid Biosciences is expecting to provide initial safety data on SGT-003 from the first couple of pediatric patients with Duchenne muscular dystrophy enrolled in the phase 1/2 INSPIRE Duchenne trial in mid-2024.

Avidity Biosciences is anticipated to initiate its phase 3 HARBOR trial assessing AOC 1001 as a treatment for patients with myotonic dystrophy type 1 in the second quarter of 2024.

As the company gains more information about the patient death, dosing in the other phase 3 trial, CIFFREO, has been paused until further notice.

Despite failing to meet the primary end point, AMO-02 achieved clinically and statistically significant benefit in various functional and objective assessments in the phase 2/3 REACH-CDM trial.

After 2 years of treatment, EDG-5506, otherwise known as sevasemten, was well-tolerated, with rapid and sustained decreases in biomarkers of muscle damage.

The executive vice president and chief research officer of the Muscular Dystrophy Association shared her reaction to the recent FDA approval of givinostat for Duchenne muscular dystrophy.

The second-year PhD student in bioinformatics at Boston University who lives with LAMA2 congenital muscular dystrophy talked about the potential impact and challenges of gene therapy in neuromuscular diseases. [WATCH TIME: 5 minutes]

The neuromuscular specialist at the Neurology & Neuromuscular Care Center discussed the pressing need to address the transition of care from pediatric to adult healthcare systems in Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]

A patient advocate within the ALS community discussed the importance of incorporating patients and their voices into clinical meetings and trial design to ultimately meet treatment goals. [WATCH TIME: 4 minutes]

Branded as Duvyzat, givinostat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.

The chief executive officer at Elixirgen Therapeutics provided commentary on the promise of the Bobcat mRNA, and the mechanism of action for mRNA-like therapies to treat Duchenne muscular dystrophy.

The president and chief executive officer of Satellos Bioscience provided insight on a new small molecule therapy, SAT-3247, in development for patients with muscular dystrophies. [WATCH TIME: 3 minutes]

A recent study presented at MDA 2024 highlighted the evolving respiratory patterns in pediatric patients with Duchenne muscular dystrophy, offering crucial insights for effective respiratory management in this patient population.

A recent analysis of data from the US National Registry presented at MDA 2024 revealed gender disparities in pediatric-onset facioscapulohumeral muscular dystrophy, with girls experiencing more severe outcomes.

At MDA 2024, Brooke Eby shared her perspective as a patient with ALS and the ways to incorporate business approaches to improving clinical care and increasing enrollment for drug trials. [WATCH TIME: 5 minutes]

In a recent phase 2 trial analysis of viltolarsen presented at MDA 2024, findings showed improvement in forced vital capacity compared with standard care in patients with Duchenne muscular dystrophy.

A new meta analysis of 3 studies presented at MDA 2024 revealed that ataluren significantly slowed the decline in muscle function for patients with nonsense mutation Duchenne muscular dystrophy.

A recent post hoc analysis of the phase 3 EPIDYS trial presented at MDA 2024 revealed significant positive outcomes with givinostat, a histone deacetylase inhibitor, among patients with Duchenne muscular dystrophy.

Over a 52-week treatment period, treatment with SRP-9001 resulted in improvements in secondary outcomes of time to rise, micro-dystrophin expression, and 10-meter walk/run.

Patients with Duchenne muscular dystrophy between ages of 6 and 24 months old demonstrated a similar safety profile on eteplirsen than those between 24 and 48 months of age.

In a real-world study, eterplirsen was safe for patients with Duchenne muscular dystrophy, with sustained or improved status in function.