AAV8-LSPhGAA for the treatment of Pompe disease seems to be safe and bioactive, according to results from a phase 1 clinical trial.
MDA Clinical & Scientific Conference 2022
Patients with Pompe disease experience a long and distressing diagnostic process and increased isolation and deterioration due to COVID-19.
Positive results have been announced for a phase 1/2 study on AT-GAA treatment for patients with Pompe disease.
A new family of adeno-associated virus (AAV) capsid variants enable the delivery of the desired gene specifically to muscles.
Results from Part A of the phase 2 MOMENTUM trial testing the safety and tolerability of SRP-5051 in Duchenne muscular dystrophy are positive.
The microRNA miR-128 is involved in the pathology of Duchenne muscular dystrophy (DMD) and could be a promising approach for therapeutics.
Deflazacort delays the progression of multiple ambulatory milestones in Duchenne muscular dystrophy (DMD) patients, compared to prednisone.
Gene editing can be effective in correcting mutations in the human genome and treating diseases such as DMD, an expert said at the annual MDA conference.
There are a number of DMD treatment options, with each demonstrating benefits but also potential risks and complications, Katherine Mathews, MD, said.
Telehealth can help patients with neuromuscular diseases who cannot reach specialized centers, even though the approach has its limitations, experts said.
Casimersen (Amondys 45) significantly increased dystrophin expression at 48 weeks after treatment initiation in patients with DMD, researchers reported.
The effect of the sweeping antibody GYM329 in combination with risdiplam in patients with SMA will be investigated in a 2-part clinical trial.
Assessment of global longitudinal strain can effectively help monitor myocardial involvement in patients with DMD, Brazilian researchers reported.
Researchers reported from an ongoing trial that the initiation of nusinersen in infants at the presymptomatic stage of SMA is beneficial.
While more drugs for neuromuscular diseases are being approved than ever before, it’s still a challenge to make those therapies available to all patients.
Pulmonary hygiene is essential to preventing illness and reducing hospitalizations rates in patients with neuromuscular disorders including DMD, an expert said at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference.
The level of expression of the MTCH2 gene may be involved in the timing and severity of cardiomyopathy in patients with DMD, according to a new study presented at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference.
Patients with Duchenne and Becker muscular dystrophies experience high rates of cognitive learning challenges, according to caregiver assessments.
Results from a phase 2 trial suggest delandistrogene moxeparvovec (SRP-9001) has a biological effect that may be clinically relevant in DMD.
Researchers joined forces to better understand the serious adverse events that occurred during trials evaluating gene therapies to treat DMD.
Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) will take center stage at the first in-person annual gathering of the Muscular Dystrophy Association (MDA) since March 2019.
The Muscular Dystrophy Association (MDA)’s 2022 clinical and scientific conference will be held March 13-16 both virtually and in person at the Gaylord Opryland Resort and Convention Center in Nashville, Tennessee. Registration is now open.
Rare Disease Advisor is pleased to offer live coverage of the MDA Clinical & Scientific Conference 2022.
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