Aaron was born with muscular dystrophy and suffered a stroke in 2023, then spent much of the following year recovering in the Midwest because of Maui's limited medical resources. "It was beyond ...

Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone ...

I recently had the opportunity to attend a Singaporean theater production titled “Supervision,” thanks to the SingHealth Patient Advocacy Network (SPAN). SPAN is a collective of patients and ...

The wolf pups, which range in age from three to six months old, have long white hair, muscular jaws and already weigh in at around 80 pounds — on track to reach 140 pounds at maturity ...

Copyright 2025 The Associated Press. All Rights Reserved. Copyright 2025 The Associated Press. All Rights Reserved. Three genetically engineered wolves that may ...

for the treatment of myotonic dystrophy type 1 (DM1), an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease ...

Distinctive traits of the dire wolf include light thick fur and muscular jaws, while they are also much larger than grey wolves. Colossal said the animals will live their lives on a continuously ...

More information: Dariusz C. Górecki et al, Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy? Gene Therapy (2025). DOI: 10.1038/s41434-025-00531-y ...

This undated photo provided by Colossal Biosciences shows a young wolf that was genetically engineered with similarities to the extinct dire wolf. (Colossal Biosciences via AP) ...

Global Duchenne Muscular Dystrophy Drugs Market value attained USD 3.9 Billion in 2025. The market will achieve USD 6.5 Billion during 2034 with a growth rate (CAGR) of 10.96% during the forecast ...

Purpose: X-linked infantile spinal-muscular atrophy (XL-SMA) is a rare disorder, which presents with the clinical characteristics of hypotonia, areflexia, and multiple congenital contractures ...

Conclusions: Previous case reports of SMA I patients with congenital heart defects did not clarify whether the cardiac malformations were coincidental. Given the respective incidences of congenitally ...