Medical trial in US offers hope to Liam

A YOUNG Kerry boy suffering from a rare condition, Battens disease, has been invited to the US for assessment of a medical treatment trial which could save his life.

Following examinations at Temple Street Children’s Hospital, Dublin, and at Great Ormond Street Hospital, London, Liam Heffernan’s results were sent to the medical team at Weill Cornell Medical Centre, New York. He will attend there in the next fortnight.

The two-year-old from Keel, Castlemaine, will undergo an intensive baseline assessment of his present condition to establish his suitability for a life-saving gene vector transfer operation later this year.

He would become the first Irish child to receive this experimental treatment, which has been provided to just over a handful of children since this trial began in August 2010.

Liam’s five-year-old sister Saoirse died from Battens disease on January 18 last. She narrowly missed out on receiving treatment last October following a rapid worsening of her condition.

Parents Tony and Mary Heffernan who launched the National Battens Disease Charity, Bee For Battens, in 2010 had previously travelled to the US hospital with Saoirse.

The couple yesterday welcomed the calling of Liam for assessment. “This is the first bit of good news which our family has received since Saoirse’s death and while we are not building our hopes too much, we are very hopeful Liam will get the opportunity to participate,” said Mr Heffernan.

Battens disease is an inherited disorder of the nervous system that usually manifests itself in childhood.

Early symptoms of the disease usually appear when parents or doctors may notice a child begin to develop vision problems or seizures.

There can also be other personality and behaviour changes, delayed speech, slow learning, clumsiness or stumbling.

Over time, affected children suffer mental impairment, worsening seizures and progressive loss of sight and motor skills. Children become totally disabled and eventually die.

The disease in infants begins between about six months and two years of age and progresses rapidly. Patients usually die before the age of five years, although some have survived a few years.

Meanwhile, Noah Coughlan, from Florida, has started a 2500-mile Battens disease Run for Research across the US, from San Diego to Florida.

Further information is available at www.battenjourney.com & www.BeeForBattens.org

The Bee For Battens Charity is a registered non-profit and volunteer-based organisation, created to support Irish Battens disease sufferers and their families. It also funds research and the participation of children in medical treatment trials.