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#NCPE

# ncpe - Sunday 10 March, 2019

'We are failing these children spectacularly': Row over drug with 'exorbitant' cost rumbles on
# Spinraza
# Spinraza

'We are failing these children spectacularly': Row over drug with 'exorbitant' cost rumbles on

Mar 10th 2019, 8:00 AM 9,231 Views 13 Comments

Negotiations, and clashes, over the price of Spinraza are ongoing behind the scenes.

# ncpe - Sunday 3 February, 2019

'You wouldn't tell a child they couldn't have chemo, but our son is being denied the medication he needs'
# Spinraza
# Spinraza

'You wouldn't tell a child they couldn't have chemo, but our son is being denied the medication he needs'

Feb 3rd 2019, 4:00 PM 19,326 Views 17 Comments

Parents of children with a rare genetic condition are fighting for access to a potentially life-changing drug.

# ncpe - Wednesday 15 November, 2017

'The system is not adequate': Government sets up new way to judge medicines for rare diseases
# Cost Effective
# Cost Effective

'The system is not adequate': Government sets up new way to judge medicines for rare diseases

Nov 15th 2017, 10:05 PM 9,584 Views 5 Comments

Many drugs for rare diseases have been ruled as not “cost-effective” under existing criteria.

# ncpe - Friday 20 October, 2017

'If I stay in Ireland, my daughter could be slipping through my fingers'
# Pku
# Pku

'If I stay in Ireland, my daughter could be slipping through my fingers'

Oct 20th 2017, 12:05 AM 29,580 Views 6 Comments

People with PKU, a rare genetic disorder, are fighting for access to medication.

# ncpe - Saturday 26 August, 2017

'In danger of snatching defeat from the jaws of victory': How tension built ahead of Orkambi deal
# Cystic Fibrosis
# Cystic Fibrosis

'In danger of snatching defeat from the jaws of victory': How tension built ahead of Orkambi deal

Aug 26th 2017, 12:05 AM 12,513 Views 11 Comments

HSE and Vertex employees were working long hours behind the scenes to secure the historic agreement.

# ncpe - Saturday 13 May, 2017

Two boys fighting for access to drug that could help them keep walking

Two boys fighting for access to drug that could help them keep walking

May 13th 2017, 8:15 PM 13,957 Views 12 Comments

Translarna, the only treatment for boys with a specific type of Duchenne muscular dystrophy, costs €400,000 per patient per year.

# ncpe - Thursday 4 May, 2017

'I'm living proof it works': Patients with severe lung condition call for access to 'life-changing' drug
# Alpha-1
# Alpha-1

'I'm living proof it works': Patients with severe lung condition call for access to 'life-changing' drug

May 4th 2017, 6:00 AM 14,626 Views 7 Comments

People are set to lose access to Respreeza at the end of the month.

# ncpe - Saturday 7 January, 2017

'If she gets another chest infection she probably won't make it, that’s the reality'
# SPINAL MUSCULAR ATROPHY
# SPINAL MUSCULAR ATROPHY

'If she gets another chest infection she probably won't make it, that’s the reality'

Jan 7th 2017, 7:25 AM 31,738 Views 16 Comments

Nellie and Lara are the only two children in Ireland with Spinal Muscular Atrophy Type 1 – an extremely rare genetic condition.

# ncpe - Sunday 27 November, 2016

Cystic fibrosis patients 'dismayed' at decision to not fund life-changing drug
# Orkambi
# Orkambi

Cystic fibrosis patients 'dismayed' at decision to not fund life-changing drug

Nov 27th 2016, 3:35 PM 16,737 Views 52 Comments

Simon Harris has said he has not yet received a decision from the HSE regarding Orkambi.