DAVID Fiant says his cystic fibrosis and the arduous therapy it required was so bad he "could no longer tell if I was living to heal myself or healing myself to live".
While waiting for a lung transplant, he was on oxygen therapy and had a six-hour daily care regime, as well as three to four weeks of antibiotic infusions a year.
Then he became one of the first people in France to take a new triple-drug treatment that has been hailed as a gamechanger for people with the rare life-threatening disorder.
"I took my first dose of the medicine one morning. By 3pm I was feeling first effects," he said.
For the first time in years he was able to shower by himself, climb 15 steps at once and above all "accompany my daughter to buy comic books", the 40-year-old said.
When Fiant's doctor examined him after he started the treatment, he could hear air circulating in his lungs. That had never happened before, he said.
Fiant's regime of care has since been drastically cut back -- and he has become the president of the French non-profit Vaincre la Mucoviscidose (Defeat Cystic Fibrosis).
While the treatment's results were "spectacular", he emphasised that it was no miracle.
"There is still no cure for cystic fibrosis," he stressed.
- Life expectancy rising -
More than 70,000 people worldwide are estimated to suffer from the inherited disease, in which a defective gene causes sticky mucus to build up in the lungs, digestive tract and other parts of the body.
It causes difficulty breathing as well as long-term complications such as infection which leads patients to have significantly shortened life expectancies.
However the triple-pill treatment taken by Fiant, which is sold as Kaftrio in Europe and Trikafta in the United States, has had a major impact since the US first approved it in 2019.
The life expectancy of people with cystic fibrosis born between 2017 and 2021 in the US is now 53, up from 38 a decade ago, according to US non-profit organisation the Cystic Fibrosis Foundation.
Kaftrio has been hailed as a breakthrough because it targets the underlying cause of the disease, repairing a mutated defect in the CFTR gene.
The treatment was approved by the European Union's medicines agency and by Britain in August 2020.
However, out of the 7,500 cystic fibrosis patients in France, only 40 percent are able to use the treatment.
It has not been approved for children under the age of six, and 15 percent of patients have a genetic profile that renders the treatment ineffective.
Around 900 patients who received transplants of organs such as lungs or livers are also ineligible.
- 'Sword of Damocles' -
Sabrina Perquis, 41, watched the arrival in France of the new treatment with "great hope".
But she had had a transplant 15 years ago and found out she was not able to take Kaftrio.
"It was a tough blow," she admitted.
"When you have had a transplant you live with a sword of Damocles hanging over your head because rejection is always possible," she added.
"We are asking not to be forgotten. Research must continue to find a solution for all patients."
Several research projects are at varying stages of development, searching for an answer for patients with rare genetic mutations.
Other questions remain, including whether the treatment completely blocks the disease's progress or merely slows it down, Vaincre la Mucoviscidose vice-president Pierre Foucaud said.
The number of cystic fibrosis patients who had an organ transplant fell from 21 to just two per quarter in France from 2019 to 2021, spurred by hopes that Kaftrio would render the operation unnecessary.
"But will these transplants merely be delayed by 10 to 15 years?" Foucaud asked. "We don't know."
Kaftrio has "opened up a tremendous hope for all patients" but more needs to be done, he added.
ETX Studio
Tue Sep 27 2022
More than 70,000 people worldwide are estimated to suffer from the cystic fibrosis, in which a defective gene causes sticky mucus to build up in the lungs, digestive tract and other parts of the body. - ETX Studio
COP29 climate summit draft proposes rich countries pay $250 billion per year
The draft finance deal criticised by both developed and developing nations.
Bomb squad sent to London's Gatwick Airport after terminal evacuation
This was following the discovery of a suspected prohibited item in luggage.
Kelantan urges caution amidst northeast monsoon rains
Kelantan has reminded the public in the state to refrain from outdoor activities with the arrival of the Northeast Monsoon season.
Former New Zealand PM Jacinda Ardern receives UN leadership award
Former New Zealand prime minister Jacinda Ardern was given a global leadership award by the United Nations Foundation.
ICC'S arrest warrants for Netanyahu, Gallant an apt decision - PM
The decision of the ICC to issue arrest warrants against Benjamin Netanyahu and Yoav Gallant is apt, said Datuk Seri Anwar Ibrahim.
KTMB provides two additional ETS trains for Christmas, school holidays
KTMB will provide two additional ETS trains for the KL Sentral-Padang Besar route and return trips in conjunction with the holidays.
BNM'S international reserves rise to USD118 bil as at Nov 15, 2024
Malaysia's international reserves rose to US$118.0 billion as at Nov 15, 2024, up from US$117.6 billion on Oct 30, 2024.
Findings by dark energy researchers back Einstein's conception of gravity
The findings announced are part of a years-long study of the history of the cosmos focusing upon dark energy.
NRES responds to Rimbawatch press release on COP29
The Ministry of Natural Resources and Environmental Sustainability (NRES) wishes to offer the following clarifications to the issues raised.
Online Safety Bill and Anti-Cyberbullying Laws must carefully balance rights and protections
The Online Safety Advocacy Group (OSAG) stands united with people in Malaysia in the fight against serious online harms.
Malaysia's inflation at 1.9 pct in Oct 2024 - DOSM
Malaysia's inflation rate for October 2024 has increased to 1.9 per cent, up from 1.8 per cent in September this year.
Saudi Arabia showcases Vision 2030 goals at Airshow China 2024
For the first time, Saudi Arabia is participating in the China International Aviation & Aerospace Exhibition held recently in Zhuhai.
King Charles' coronation cost GBP 71mil, govt accounts show
The coronation of Britain's King Charles cost taxpayers GBP72 million (US$90 million), official accounts have revealed.
Couple and associate charged with trafficking 51.9 kg of meth
A married couple and a man were charged in the Magistrate's Court here today with trafficking 51.974 kilogrammes of Methamphetamine.
PDRM to consult AGC in completing Teoh Beng Hock investigation
The police may seek new testimony from existing witnesses for additional insights into the investigation of Teoh Beng Hock's death.
Thai court rejects petition over ex-PM Thaksin's political influence
Thailand's Constitutional Court rejects a petition seeking to stop Thaksin Shinawatra from interfering in the running the Pheu Thai party.
Abidin takes oath of office as Sungai Bakap assemblyman
The State Assemblyman for Sungai Bakap, Abidin Ismail, was sworn in today at the State Assembly building, Lebuh Light.
UPNM cadet officer charged with injuring junior, stomping on him with spike boots
A cadet officer at UPNM pleaded not guilty to a charge of injuring his junior by stomping on the victim's stomach with spike boots.
How Indian billionaire Gautam Adani's alleged bribery scheme took off and unraveled
The indictment was unsealed on Nov. 20, prompting a $27 billion plunge in Adani Group companies' market value.
Elon Musk blasts Australia's planned ban on social media for children
Several countries have already vowed to curb social media use by children through legislation, but Australia's policy could become one of the most stringent.