脑部疾病与治疗

抽象的

Editorial Note: Brain disorders & Therapy

Sarilla Gowthami

Neuromuscular diseases have a considerable burden on patients, their caregivers, and healthcare systems. These diseases are largely genetic, degenerative, and life-threatening or life-altering. The dystrophy Association (MDA) has been at the forefront of research, therapy development, and early intervention for neuromuscular disease since its foundation in 1950. The MDA Clinical & Scientific Conference, held on March 22–25, 2020, in Orlando, Florida, will mark the 70th anniversary of the MDA. Huge progress has been made in recent years, but the sector has reached a crossroads—strong advocacy programmes from the MDA and other stakeholders are going to be essential to continue the success stories of neuromuscular research and to make sure the readiness of systems to translate these breakthroughs to clinical practice.