As Prescribed: How treatment for muscular dystrophy is continuing to advance

SAN FRANCISCO (KCBS RADIO) - A quarter of a million Americans are living with muscular dystrophy or a related neuromuscular disorder. It's the reason September has been set aside to recognize the challenges associated with the illness and the progress that's been made in treating it.

Neuromuscular diseases affect the nerves that control voluntary muscle movement as well as nerves that relay sensory information to the brain, explained Dr. Pritikanta Paul, a neurologist with UCSF Health.

"They are not working properly in communication. So that's where patients present with muscle weakness or muscle wasting. And in severe cases, patients can present with paralysis. If the neuromuscular condition also involves nerves, patients can have complaints of loss of sensation or pain," Dr. Paul told KCBS Radio's Bret Burkhart on this week's episode of "As Prescribed."

While some of the conditions are genetic and can be inherited, other forms are acquired and immune mediated.

"It can be your immune system, unfortunately, attacking different parts of the nerve or the muscle and causing the manifestations of that neuromuscular disease," said Dr. Paul.

The good news is that there has been huge growth in terms of diagnosing and treating the conditions, including genetic testing that can more confidently tell what type of genetic disease, neuromuscular disease or muscular dystrophy patients have.

"We have also made some improvements in terms of monitoring disease, including different imaging techniques of muscles. For example, we use MRI and ultrasound, less invasive to diagnose or at least monitor disease. So that's more in the diagnostic landscape," said Dr. Paul. "Also in terms of the therapeutic options, there has been a huge growth for neuromuscular diseases, both genetic and acquired diseases."

That includes newer treatments that can lead to better prognosis and in some cases, full recovery to normal functioning.

"Several treatments have been approved and there are ongoing trials related to muscular dystrophies like Duchenne muscular dystrophy. There is new treatment for a rare genetic form of ALS or amyotrophic lateral sclerosis, which is also a newer treatment available for this particular genetic form of ALS. In terms of acquired neuromuscular disorders, there also has been significant growth in terms of therapeutic options available for physicians," said Dr Paul. "So there has been like a huge growth and very exciting times for neuromuscular disorders or patients with neuromuscular disorders."

And medicine is still evolving, to the point where Dr. Paul sees a future in which we might actually be able to prevent neuromuscular diseases -- and it's not that far away.

"I feel in my understanding or experience, say five or 10 years from now, we will have more information where we can actually stop from the actual manifestation of the disease," he said.

Listen to this week's "As Prescribed" to learn more. You can also listen to last week's episode to hear about new research revealing some surprising effects of "pelvic yoga," here .

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“As Prescribed” is sponsored by UCSF.