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SMA Patient Recalls Best Christmas Present Ever

Patient Lisa Velez-Batista with Dr. Lauren Elman

Lisa Velez-Batista remembers tiring easily as a child and not being able to keep up with her friends. She always questioned what was wrong, but she met all her growth milestones, so her concerns were never addressed.

Things continued to worsen until, at age 20, she fell in the street in front of a bus and couldn’t get up. That incident finally led to a devastating diagnosis: spinal muscular atrophy (SMA).

“I was told there was no treatment, that it was progressive, and I would be wheelchair-bound in 10 years,” says Velez-Batista, who is now 56. “I had just completed my associate’s degree but decided to put my bachelor’s on hold. Soon after I had two children. I wanted to live life while I could.”

Velez-Batista had SMA-3 and was fortunate that her disease progressed slowly. She eventually returned to college and then worked full-time as a speech pathologist before retiring early due to a knee injury. But you can’t outrun SMA.

“I’ve become much weaker over the years, and I now have to use a scooter if I need to walk any distance,” she says. “I had resigned myself to a slow deterioration.”

Things changed on Christmas Eve 2016 when her cell phone began blowing up with phone calls and text messages. “Did you hear?” friends and relatives asked. The FDA had just approved Spinraza, a drug that can slow progression of SMA and, in some cases, improve strength in patients.

“I had heard of this drug being tested in children, but I didn’t think adults would get it,” she says. “I had the first of my four loading doses at Penn in October 2017 and am now on my third maintenance dose.”

Velez-Batista says she almost immediately felt “lighter” after the first dose. That feeling was backed up by measurable improvements.

“I started physical therapy and soon I could do small things I couldn’t do before, like bending my knees back when I lay on my stomach or ripping a piece of folded up paper,” she says. At a recent appointment, Penn neurologist Lauren Elman, MD, even detected a reflex in Velez-Batista’s right arm, something that hadn’t happened in years.

The most important thing is that the disease is not progressing,” says Velez-Batista. “It’s not a cure, but I feel stronger and if I’m not getting worse that’s a great thing.”

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