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Batten disease
This page shows the latest Batten disease news and features for those working in and with pharma, biotech and healthcare.
NICE approves BioMarin’s Brineura after initial setback
First treatment for rare disease. BioMarin and NICE had a rocky start with Batten disease therapy Brineura, but the two have now come to an agreement. ... The disease, known as neuronal ceroid lipfuscinosis type 2 (CLN2), is a very rare inherited
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BioMarin ticks over in Q2 ahead of gene therapy filing
A second-generation treatment for rare disease phenylketonuria (PKU), the newly launched drug reported $18.8m in sales. ... BioMarin will also hope to extend the commercial success of Brineura, its Batten disease therapy.
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Amicus forms gene therapy manufacturing partnership with Catalent
Amicus has one rare disease medicine already on the market, Galafold for Fabry disease, with an enzyme replacement therapy (AT-GAA) for Pompe disease in its late stage pipeline, to which ... capabilities. Thermo Fisher will supply Amicus with immediate
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NICE knocks back BioMarin’s Batten disease drug
The committee initially agreed that Brineura shows substantial short-term benefits in slowing down the rate of Batten disease progression in draft guidance, but said BioMarin was unable to price the ... Results from a phase 1 and 2 clinical trial show
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CHMP backs two rare disease therapies at April meeting
BioMarin got a green light for Brineura (cerliponase alfa), an enzyme replacement therapy for the ultra-rare neurodegenerative disease neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease that ... There is no approved drug to treat the
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Genomic medicine: collaborating for diverse data
In fact, it is the disease area that has made the most progress in developing personalised medicine. ... However, there have been recent developments in the use of gene therapy for spinal muscular atrophy (SMA) and trials are underway for
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Rare disease drug development – real-world evidence innovations with historical controls
By Will Maier. Rare disease drug development poses special challenges that can be overcome by using real-world evidence (RWE). ... a common form of Batten disease.
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How understanding the rare disease patient’s lived experience creates better health communications
Children like Noah Van Houtan, and his sister Laine – who both had CLN2 Batten Disease. ... With the co-operation of the VanHoutan family, the film describes their journey with CLN2 Batten Disease.
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Bedrock mark International Batten Disease Awareness Day 2022 with Noah's Story
Batten Disease is a group of fatal genetic disorders. There are 13 types. ... Hope4Bridget and the story that the VanHoutan’s share about their family’s journey with CLN2 Batten Disease.
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Shining a light on rare diseases with ‘Noah’s Story’
In recognition of Rare Disease Day, Bedrock share 4 minutes of a true story you won’t want to miss. ... Sadly about 75% of rare diseases affect children like Noah who had CLN2 Batten Disease, and one in three children with a rare disease won’t live
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Bedrock to raise awareness of Rare Disease Day 2022 with film 'Noah's Story'
Children like Noah – who had CLN2 Batten Disease. The film will premiere on Rare Disease Day, Monday February 28th. ... Hope4Bridget and the story that the VanHoutan’s share about their family’s journey with CLN2 Batten Disease.
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Bedrock Healthcare Communications
Content strategy and execution. Disease awareness campaign and fulfilment. Digital strategy and engagement. ... Bedrock mark International Batten Disease Awareness Day 2022 with Noah's Story.
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- Based on research with HCPs in the UK, USA and France, Graphite Digital’s latest whitepaper sheds light on HCP perceptions of pharma-owned digital channels, and what organisations should be prioritising...
