Afib: Causes, Symptoms, and Treatment Options
SCAD: Sisters Had Heart Attacks Just Days Apart Due To Rare Condition
Rebecca and Angharad had heart attacks three days apart
Two sisters who had heart attacks just days apart have said more should be done to raise awareness of a condition that can be fatal.
Rebecca Lewis, 48, had a spontaneous coronary artery dissection (SCAD), when a tear appears in the wall of a coronary artery.
Although not conclusive, it is likely that her sister Angharad experienced the same thing three days later.
SCAD is a rare condition that cannot currently be predicted or prevented.
The majority of cases affect women in their 40s or 50s or those who have recently had a baby.
Rebecca, a teacher from Cardiff, was marking work in her classroom last November when she felt huge pressure on her chest.
The school's quick-thinking head teacher took Rebecca to the University Hospital of Wales in Cardiff, where she was told she had had a heart attack.
An angiogram a few days later showed that it had been caused by a SCAD.
What is SCAD?
A SCAD happens when the inner layers of a coronary artery tear away from the outer layer, restricting blood flow.
In some cases this can lead to a heart attack or cardiac arrest.
Symptoms can include chest pain, tightness or pain in the arms, neck, jaw, back or stomach; feeling dizzy or lightheaded; feeling tired or out of breath; nausea, and feeling sweaty or clammy.
Rebecca Lewis, 48, said she felt huge pressure on her chest while at work one day
Rebecca said she was lucky that the consultant who treated her had previously worked at Glenfield Hospital in Leicester, which is a leading centre for research into SCAD.
"The consultant that actually did my angiogram... Had been trained to know what SCAD looked like on an angiogram and was able to pick it out with me," she said.
The morning after visiting her in hospital, Rebecca's sister Angharad also had a heart attack. An angiogram suggested she too had suffered a SCAD.
Angharad was treated at a different hospital, but Rebecca said she was adamant that Angharad should be checked for a SCAD.
Angharad had a heart attack three days after her sister
"They were happy to discharge me," Angharad recalled.
"So I was very fortunate that I had a big sister looking after me saying 'you need to get this test, you need to mention what has happened to me and what they found'.
"So, eventually, the consultants spoke to each other and I was sent down to Cardiff. If it wasn't for Becky, I would've been sent home not knowing why I'd had my heart attack."
There are currently 2,000 patients helping the research at Glenfield Hospital.
Prof David Adlam, who leads the research, said many cases go undiagnosed.
"We try to encourage our colleagues across the health services, who will be potentially seeing patients who might have SCAD, to think of it. Because if you're not thinking of it, then you may miss it," he said.
"When you have a conventional heart attack... Generally it's caused by a fixed narrowing in the coronary artery, and we often treat that by stretching open that narrowing and inserting a stent.
"In SCAD patients we do the opposite," he added.
"We're trying to manage the artery to let it heal by itself. And the reason for that is that it's a different disease. It has a different underlying cause."
Rebecca and Angharad are still coming to terms with what happened to them.
"It's affected the whole family," said Angharad.
Angharad says the experience has affected the whole family
"I've been very fortunate to have support from friends. They've been coming here just because they are aware that I'm a bit nervous about doing things and being out and about."
Rebecca said the experience had knocked her confidence.
"It caused a lot of distress when it happened," she said.
"It was the last thing I was thinking at 48 years of age that I'd be having a heart attack."
Research into SCAD is currently funded by the Beat SCAD charity, although the Scottish government is also running a pilot project.
Prof Adlam hopes the Welsh government will do the same.
"We would very much like to work with our friends and colleagues in Wales to ensure that these patients are properly cared for," he said.
"It's really important to raise awareness," Angharad added.
"At the moment we are saying it's very rare, but if we're looking at my experience, maybe it's going undiagnosed. Becky came home with a diagnosis, with a label. Fortunately, I've ended up with the same, but it could have been very different."
The Welsh government said: "We expect NHS Wales to deliver care for acute coronary syndrome in line with professional guidelines from organisations such as the National Institute for Health and Care Excellence (NICE).
"For more rare cardiac conditions, NHS Wales is prepared to work with counterparts across the UK on research."
'I was told I'd die if I had a baby'
Heart attack inequality 'costing women's lives'
Women at higher risk of heart attack misdiagnosis
Ractigen Therapeutics Announces FDA Orphan Drug Designation For RAG-21 For The Treatment Of ALS
NANTONG and SUZHOU, China, Nov. 19, 2024 /PRNewswire/ -- Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing innovative therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy targeting the FUS gene, for the treatment of amyotrophic lateral sclerosis (ALS).
RAG-21 is an siRNA therapy specifically designed to target FUS-ALS, one of the most aggressive subtype of ALS. By leveraging RNA interference (RNAi), RAG-21 safely and effectively reduces FUS protein levels, addressing the root cause of motor neuron degeneration in FUS-ALS. Preclinical studies have demonstrated its potent efficacy and safety, mitigating FUS cytoplasmic mis-localization and aggregation. As a novel RNA-based therapy, RAG-21 offers renewed hope for improving outcomes in patients suffering from this challenging form of ALS, which currently lacks effective treatment options. This designation marks our second FDA ODD for treating ALS, following RAG-17 for SOD1-ALS, reflecting our commitment to conquering ALS.
Orphan drug designation is granted by the FDA to a drug intended to treat a rare disease or condition, which generally affects fewer than 200,000 individuals in the United States. The designation provides companies with incentives including a 25% tax credit on qualified clinical trials, a seven-year marketing exclusivity and a waiver of the New Drug Application fee.
"The FDA's Orphan Drug Designation for RAG-21 underscores the critical need for innovative therapies targeting ALS, particularly for patients with FUS mutations," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "FUS-ALS represents one of the most severe and rapidly progressing subtypes of ALS, with no curative treatments currently available. We are committed to developing innovative therapies like RAG-21 to provide meaningful treatment options for patients with ALS and other life-threatening rare diseases."
About RAG-21
RAG-21 utilizes RNA interference to selectively reduce FUS mRNA transcripts. This mechanism prevents the production of toxic FUS proteins and addresses the underlying pathology of FUS-ALS. Delivered via the SCAD™ platform, RAG-21 achieves targeted, efficient, and durable gene knockdown within the CNS. Preclinical data highlight its potential to mitigate motor neuron degeneration and improve disease outcomes, offering hope for patients with this aggressive ALS subtype.
About ALS and FUS Mutations
ALS is a devastating neurodegenerative disease with no cure, significantly impairing patients' quality of life. Most patients succumb to respiratory failure within 2-5 years of diagnosis. Mutations in the FUS gene are associated with a particularly aggressive form of ALS, characterized by early onset and rapid progression. These mutations lead to toxic protein accumulation, mis-localization, and the formation of abnormal inclusions in neurons, ultimately lead to motor neuron degeneration.
Despite progress in ALS drug development, effective treatments remain elusive, especially for FUS-ALS. Current therapies, such as Riluzole and Edaravone, offer only modest benefits and are not specific to FUS-ALS. RNA-based therapeutics, including siRNA, represent a promising strategy by directly targeting disease-driving genes like FUS to address the root cause of the disease.
About Ractigen Therapeutics
A leader in small activating RNA (saRNA) drug development, Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing RNA activation (RNAa) mechanism to up-regulate endogenous gene expression. This innovative approach involves saRNA targeting specific genes to enhance transcription, thereby restoring normal protein functions. Ractigen's cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation. For more information, please visit our website at www.Ractigen.Com.
View original content:https://www.Prnewswire.Com/news-releases/ractigen-therapeutics-announces-fda-orphan-drug-designation-for-rag-21-for-the-treatment-of-als-302309985.Html
SOURCE Ractigen Therapeutics
Comments
Post a Comment