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SNP MP Marion Fellows with her granddaughter Saoirse, diagnosed at the age of three weeks.
Families have welcomed the agreement that will allow eligible patients in Scotland to access two essential drugs for the treatment of cystic fibrosis.
A five-year agreement was reached between the Scottish Government and the pharmaceutical company Vertex regarding the use of Orkambi and Symkevi.
The drugs improve lung health but were rejected for use of the NHS last month because they were not considered profitable.
The drug normally costs £ 100,000 per patient per year.
The Scottish Government has stated that it has obtained a "confidential rebate" from the manufacturer.
Patients from other parts of the UK are still not able to access treatment, as activists claim the end of the "lottery by zip code".
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Marion Fellows, SNP MP for Motherwell and Wishaw, whose three-year-old granddaughter, Saoirse, is suffering from cystic fibrosis, said it was "a great moment" for them. patients and their families, who had been "implacable and inspiring" in their campaign.
Ms Fellows said: "I am so happy that so many families across Scotland are benefiting from these new medicines, and hundreds of people will be able to live longer and more fully with this decision.
"The Scottish Government has never stopped discussing and is leading the way in the treatment of cystic fibrosis.
"But cystic fibrosis knows no boundaries, the British government must follow the example of the Scottish government and make it accessible to people with the disease in England."
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Medications Can Reduce Hospitalizations by Stabilizing Pulmonary Health
Cystic fibrosis is a genetic disease that shortens life and causes fatal lung damage. It affects about 10,400 people in the UK, including about 900 in Scotland.
Only about half of those affected live up to 40 years.
NHS Scotland estimates that one in 24 Scots has a CFTR mutation which, if carried by both parents, would lead to the birth of a child with cystic fibrosis.
Glasgow's mother, Gail Gilmour, whose son has cystic fibrosis, tweeted that there were "no words" to thank those who helped secure the transaction for Scottish patients.
Others have expressed hope that this would help advance the negotiations for England, Wales and Northern Ireland.
Earlier this year, activists called on the British government to use its powers to break the stalemate by making Orkambi available on the NHS in England.
Vertex has refused a 500 million pound bid on this drug for five years – which has been described as the "biggest commitment" ever made by the NHS in England.
Affected people want other pharmaceutical companies to be invited to make a cheaper version.
The UK Department of Health said its approach was still to urge Vertex to accept NHS England's "generous offer".
"Historical moment"
The Scottish Medicines Consortium (SMC) rejected the current use of the drug in August, stating that uncertainties remain about the long-term health benefits of Orkambi and Symkevi over their costs.
David Ramsden, chief executive of the Cystic Fibrosis Trust, said the deal was a "defining moment" for the hundreds of people with cystic fibrosis and their families in Scotland.
"This breakthrough is a victory for perseverance and enduring hope.This means that 350 eligible people living in Scotland will have access to medicines that stabilize the health of their lungs and reduce the number of admissions to the hospital. . "
He added that trust would continue to campaign for access to medicines in England, Wales and Northern Ireland, adding: "The success of Scotland must now be replicated across the Kingdom. United without further delay. "
What are the Orkambi and Symkevi?
Since 2015, the drug Orkambi is licensed for the treatment of cystic fibrosis in patients aged two years to adulthood, carrying a specific genetic mutation called F508del.
It was not available on the NHS except for some people for humanitarian reasons.
Symkevi is used to treat the same mutation in patients aged 12 years and older.
The mutation causes the production of an abnormal protein that disrupts the transport of water and chlorides in the body.
Clinical trials have shown that Orkambi improves lung function and respiratory symptoms in people with cystic fibrosis.
This is the first of a series of drugs that have been developed. It is expected that new drugs will be even more effective.
Scottish Secretary of State for Health, Jeane Freeman, said this "fantastic news" would allow patients with cystic fibrosis to "live longer".
"The agreement was reached after lengthy discussions between the Scottish Government and Vertex Pharmaceuticals and means that the drugs will now be made available to NHS patients in Scotland, subject to a confidential discount," she added. .
Vertex is also committed to collecting real-time drug data to facilitate future submissions to the MSC.
Ludovic Fenaux, senior vice president of Vertex, said: "We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way in which we have worked together to find this access solution.
"This means that about 400 eligible cystic fibrosis patients in Scotland now have access to Orkambi and Symkevi."
Some Scottish patients already had access to Orkambi and Symkevi through the Level 2 Peer-Reviewed Clinical System (PACS Level 2), which allows doctors to request access on behalf of each patient.
A spokesman for SMC said the pharmaceutical industry was responsible for marketing their medicines at a fair price.
"MSC members make these difficult decisions based on extensive clinical experience and take into account all available evidence, including that provided by patient groups." We hope to receive further evidence from Vertex on the subject. 39 impact of these drugs following the agreement that they signed with the Scottish Government ".
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