SAN RAFAEL, Calif., June 1, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the European Commission (EC) has granted marketing authorization for Brineura™ (cerliponase ...
CLN2 Batten disease is a rare, inherited, fatal disorder that primarily affects the nervous system as a result of a failure to produce enough of the enzyme TPP1. The signs and symptoms of this ...
Four-year-old Noah VanHoutan seemed to be getting better. The seizures that shook his body and rattled his parents’ nerves were less frequent with a special diet and medication. His balance was still ...
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Treatment to preserve sight in children with rare genetic disorder shows promise in clinical trial
A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialed by clinicians and researchers at UCL and Great Ormond Street Hospital (GOSH). The study, ...
Downers Grove girl dies of rare disease one year after FDA approves treatment her parents fought for
Less than a year after the U.S. Food and Drug Administration approved the first drug to treat her rare disease, a Downers Grove girl died at home Wednesday with her parents, who spent nearly a decade ...
SAN RAFAEL, Calif., July 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the company's supplemental Biologics ...
WEBVTT >> READY, SET, GO.JEAN: MATTY IS A TRAILBLAZER ATJUST SEVEN YEARS OLD, PAVING THEWAY FOR OTHER CHILDREN TORECEIVE THE TREATMENT HE'S BEENGETTING IN A CLINICAL TRIAL.THE FDA APPROVAL BROUGHT ...
Most people haven’t heard of the CLN2 Batten Disease, an illness that ravages the motor skills, speech and vision of its host. Those who have the illness – mostly children – have a greatly shortened ...
A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects.
A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects.
A fundraiser for a rare neurodegenerative disorder affecting a four-year-old girl with a connection to racing was highlighted during the 2025 Indianapolis 500. Helen’s Pink Sky Foundation was featured ...
SAN RAFAEL, Calif., April 27, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) approved Brineura™ (cerliponase alfa) to ...
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