READER’S DIGEST
May• 2019 | 125would learn to feed himself and say
“Bee”, his name for his grandmother.
Then came “Da”, for Jeff.
The Beishes would need to be cau-
tiously optimistic. No one had ever
survived Batten disease. They tried
to think like scientists: incremental-
ly, with judicious notions of progress.
“I’ve heard of kids who can now walk
30 steps and kids who can sit up who
couldn’t before,” Hollie told me.
Dr de los Reyes described Brineura
as “a treatment until we find a cure.”
She told me that she was starting an
extension study of the trial to examine
the long-term safety and effectiveness
of Brineura. BioMarin is also studying
whether children under three could
be effectively treated before they ever
showed symptoms of CLN2. At Rush
University in Chicago, researchers
have been investigating therapies
that could treat TPP1 deficiencies with
pills already approved for addressing
other medical conditions.
For its part, BioMarin is scaling
up production of Brineura. It’s also
investigating the treatment as a
model for other direct-to-brain care,
which could lead to breakthroughs
for patients suffering from other rare
neurological diseases.Hollie says Conner is continuing to
progress. A recent MRI scan showed
no additional brain atrophy. Seizure
activity is stable, so his medication has
been reduced.
He can now climb on the couch and
walk better in his gait trainer. He is
babbling more and making different
sounds. His tremor has improved and
he can now reach for things and grasp
them more accurately.
ADAPTED FROM THEATAVIST MAGAZINE (NO. 74), © 2018 BY AUTOMATTIC INC., MAGAZINE.ATAVIST.COMTOWN SHUTS DOWN FOR GAMESeven thousand people descended on the town of Ashbourne,
England, recently to play the Royal Shrovetide Football Match- a centuries-old game in which the rules are hazy and the
goals 4.5km apart. Teams tap the ball three times on stone
plinths that act as goals. Played from 2pm to 10pm, goals are
rare because of the size of the pitch. Due to the large
numbers taking part, local shops closed early and
boarded up as if preparing for a riot.
REUTERS