Forty years waiting for a cure: ALD gene therapy trial shows early promise

adrenoleukodystrophy-Ethan-Emily
A small piece of notepaper, folded twice, sits tucked in a slot of the secretary desk in the living room. Every so often, I pull it out, read it, then reread.

Addressed to my mom, the paper has a question and two boxes, one “yes” and one “no,” written with the careful precision of a 7-year-old.

I am sad of Ethan. You too?

A check marks the box.

— Yes. Yes, I am sad too.

Read Ethan’s story on our Vector blog and learn more about the ALD gene therapy trial.

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From ear infections to ear tubes: A tale of 2 tots (in texts)

ear infectionsMeet Henry and Atticus — two toddlers with much in common. Both little boys live near Boston with their parents and are happy, smiley babies … most of the time. But sometimes, they aren’t quite so joyful.

Henry and Atticus have suffered chronic ear infections in infancy, and their conditions persisted to the point at which their pediatricians recommended ear tubes. Their mothers — Erin Jemiola (Henry) and Katie Monroe (Atticus) — share their text messages as they coped with their sons’ ear infections and made the decision to have ear tube surgery.

 

Katie-50pxHelp! I’m not sure if I should take Atticus to the pediatrician. He’s been tugging on his ears a lot. Otherwise, he seems fine. 

 

 

 

You may want to. Henry had similar symptoms, plus fussiness and restless sleep, after I went back to work in September. It turned out he had a double-ear infection and fluid in his ears. Erin-50px

 

 

 

 

 

 

Katie-50pxYou were right! His pediatrician put him on antibiotics. I hope it clears up. 

 

 

 

 

Good luck. Keep me posted. Henry has had three double ear infections in the last few months. :( Erin-50px

 

 

 

 

Katie-50pxI’m sorry to hear that. Atticus had another ear infection when I brought him in for his 9-month well-baby check. Dr. Ryan is going to try a stronger antibiotic. 

 

 

 

 

Dr. Sax has prescribed amoxicillin and augmentin, but Henry keeps getting colds and ear infections. Erin-50px

 

 

 

 

Katie-50pxDr. Ryan referred us to Dr. Adil at Boston Children’s Hospital. I’m really worried. Atticus’ audiology screening showed he has significant hearing loss. She thinks Atticus should get ear tubes

 

 

 

What a coincidence! We just took Henry to Dr. Adil. She thinks he might be a candidate for ear tubes, too, but said he’s a little too young for surgery. Usually, they wait until a baby has three months or more of fluid in the ear with hearing loss, three ear infections in six months or four infections in one year. We’ll see what happens in January. Fingers crossed! Erin-50px

 

 

 

 

 

 

 

 

Katie-50pxWe scheduled Atticus’ surgery at Boston Children’s at Waltham. I’m a little nervous about the anesthesia. 

 

 

 

When we went back to Dr. Sax. Henry had another ear infection. We decided it was clear he should get the tubes. I was nervous about the anesthesia, too. But we’ve done our homework and learned how this will help Henry. And Boston Children’s does 100 ear tube surgeries every week. Now, I’m feeling comfortable. Erin-50px

 

 

 

 

 

 

Katie-50px Atticus just had his surgery. It went well. I’m pretty sure it was harder on us as parents than him. He was a little drowsy when he woke up, but he already seems more verbal than he was before. 

 

 

 

 

 

That’s such good news! Erin-50px

 

 

 

 


Katie-50px
It is. I’m still worried about his hearing loss. We’ll find out if it was temporary when he sees the audiologist. Dr. Adil feels confident it will resolve. 

 

 

 

Let us know. Erin-50px

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Atticus’ post-surgery hearing test showed he no longer has hearing loss.

 

 

jemiola_erin_henry_crop
Henry has not had an ear infection since his ear tube surgery.

Henry’s surgery went great! He’s always been a happy boy, but we’ve seen such a difference — he’s more responsive and talkative! We made the right decision :) 
Erin-50px

 

 

 

 

 
 

 

What are the benefits of ear tubes?

Ear tubes, or myringotomy tubes, are placed in a child’s ear during a brief outpatient surgery called tympanostomy. These small tubes are most often placed because of persistent fluid in the middle ear, recurrent ear infections or ear infections that persist despite antibiotic treatment. Potential benefits of ear tubes includes improved hearing, reduced fluid in the middle ear and and reduced incidence of ear infections.

 

Find out about services for your child at Boston Children’s at Waltham.

 

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Spring is here! Tick and Lyme disease prevention tips

Kids bike ridingSpringtime temperatures and warmer, sun-filled days are synonymous with outdoor play. Kids of all ages shed their winter jackets; break out their tricycles, bicycles and more, and head outside for playtime.

But outdoor activities are also synonymous with tick exposure.

So how can you prepare your family for the tick season?

The Boston Children’s Hospital’s Director of Infectious Disease Outpatient Practice, Dr. Catherine Lachenauer offers tips on how to avoid tick exposure and steps to take if your child gets a tick bite.

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Less is more: Second opinion spares Sam from risky invasive surgery

tethered spinal cordLike many moms, Brenda Jackson worried about the transition to middle school for her son Sam. He had had mysterious wetting accidents ever since potty training. Doctors had diagnosed an overactive bladder and tried everything — behavioral interventions, medications and dietary changes.

Nothing worked. Fortunately, Sam attended a small Montessori school where all the kids knew and accepted each other.

But as he was getting ready for fifth grade, the specter of middle school, and the teasing that comes with early adolescence, loomed. “That’s a new ball game,” says his Brenda. “We knew we had to take care of this.”

Because Sam also had constipation, the urologist at his Midwestern children’s hospital sent him to a gastroenterologist to see if addressing it would reduce Sam’s wetting accidents. The urologist put him on a medication meant to stop the spasms that were making him lose bladder control. It didn’t work.

It was such a relief to know that Sam was able to have a less invasive procedure. Boston Children’s made a very stressful process easier and had the confidence to take care of it.

Sam’s doctors and parents continued to search for answers. A dimple on the boy’s lower back suggested something wrong with his spinal cord, so Sam was referred for an MRI of his spine.

“We got a call the next morning that Sam had a thoracic syrinx [a collection of fluid inside the spinal cord’s central canal] and that we needed to see a neurosurgeon,” says Brenda. “The neurosurgeon at our hospital had never seen one.”

The local radiologist and neurosurgeon thought the fluid was compressing the nerves that controlled Sam’s urinary tract.

After two more MRIs, the doctors recommended surgery for the syrinx. It would involve inserting a shunt (tube) inside Sam’s spinal cord to drain the fluid into his abdomen — a highly invasive operation that carries a risk of causing paralysis. Plus, shunts often fail over time and have to be replaced.

Something didn’t ring true for Brenda. Her skepticism grew. She researched Sam’s condition and found few pediatric hospitals had published anything about it. One of those that did was Boston Children’s Hospital.

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