Becoming a Scholar and a Mother: Reflections from a Student with LGMD

Pearl Burgin has faced her share of setbacks as she navigated life with limb-girdle muscular dystrophy, motherhood and higher education as an adult student. But, as she writes in this reflection on going back to school, “I am fortunate to have found a way to become a mom and a scholar. If anyone else is wondering if getting an education while living with a neuromuscular disease and raising a family is possible, I hope they consider their situation, review their options and find resources that will help. Remember not to let doubt hold you back from becoming all that you want to be. Just do what works for you. I challenged myself, worked hard, and it’s paying off. Now for the next goal: grad school.”

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Seeing the World Thanks to MDA Shamrocks: Meet Gabrielle, the Traveling Shamrockstar

Fourteen-year-old Kentucky native Gabrielle’s favorite subject is geography, which is appropriate when you have an insatiable desire to travel. Gabrielle, who has spinal muscular atrophy (SMA) Type 2, has already seen a great deal of the world, including Europe, the Caribbean and the western United States. This year, Gabrielle is featured as a “Shamrockstar” on platinum MDA Shamrocks at retailers across the country, a country that Gabrielle has seen extensively in her travels with her family.

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FirST-4-LEMS Study Seeks Participants

Researchers are looking for people with Lambert-Eaton myasthenic syndrome (LEMS) to participate in a phase 3 clinical trial, being conducted by Catalyst Pharmaceuticals, to test the investigational drug amifampridine phosphate. The Firdapse Strength Trial for LEMS, or FirST-4-LEMS, study is designed to evaluate the effectiveness of amifampridine phosphate in controlling, reducing and/or eliminating symptoms of . . .

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CMS Study Seeks Participants

Researchers are looking for people with congenital myasthenic syndrome (CMS) to participate in a phase 3 study being conducted by Catalyst Pharmaceuticals to test the experimental drug amifampridine phosphate.

Amifampridine phosphate is a potassium channel inhibitor designed to cause greater stimulation of muscle by prolonging nerve signals and is expected to help treat muscle weakness in people with CMS. The drug has shown promising results in treating patients with a related disorder called LEMS.

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Boy with juvenile dermatomyositis scores a touchdown

For more than 60 years, firefighters have teamed with MDA to fight back against muscular dystrophy, ALS and related life-threatening diseases.

For one firefighter, the cause became very personal when his son, Chandler Mallen, was diagnosed with juvenile dermatomyositis, a neuromuscular disease that involves inflammation of the muscles. When Chandler asked his father — Scott, a firefighter who has collected donations for MDA’s Fill the Boot program for almost 25 years — if he’d lose the use of his legs, he didn’t wait for an answer.

“Even if I do,” Chandler said, “I’ll never stop trying.”

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