Latest news with #Dravet
Associated Press
6 days ago
- Business
- Associated Press
Stoke Therapeutics to Present at the Jefferies Global Healthcare Conference
BEDFORD, Mass.--(BUSINESS WIRE)--May 29, 2025-- Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today announced that Interim Chief Executive Officer Ian F. Smith will present at the Jefferies Global Healthcare Conference on Wednesday, June 4, 2025, at 1:25 p.m. ET. A live webcast of the presentation, along with an archived replay, will be available in the Investors & News section of Stoke's website at About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is expected to enter Phase 3 development in 2025. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for the Company's proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit View source version on CONTACT: Stoke Media & Investor Contacts: Dawn Kalmar Chief Communications Officer [email protected] 781-303-8302Doug Snow Director, Communications & Investor Relations [email protected] 508-642-6485 KEYWORD: MASSACHUSETTS UNITED STATES NORTH AMERICA INDUSTRY KEYWORD: SCIENCE BIOTECHNOLOGY RESEARCH PHARMACEUTICAL HEALTH FDA GENETICS CLINICAL TRIALS SOURCE: Stoke Therapeutics, Inc. Copyright Business Wire 2025. PUB: 05/29/2025 07:30 AM/DISC: 05/29/2025 07:31 AM
Business Wire
6 days ago
- Business
- Business Wire
Stoke Therapeutics to Present at the Jefferies Global Healthcare Conference
BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today announced that Interim Chief Executive Officer Ian F. Smith will present at the Jefferies Global Healthcare Conference on Wednesday, June 4, 2025, at 1:25 p.m. ET. A live webcast of the presentation, along with an archived replay, will be available in the Investors & News section of Stoke's website at About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is expected to enter Phase 3 development in 2025. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for the Company's proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit
Yahoo
20-05-2025
- Health
- Yahoo
Lewisham mum and five-year-old son's challenge for rare epilepsy condition
A mum and her five-year-old son with a rare form of epilepsy are taking on a challenge to walk, run, or cycle 2k every day in May. Reniece Thomas, from Lewisham, is participating in the #EveryDayinMayForDravet challenge to raise funds and awareness for Dravet Syndrome, a severe form of epilepsy that affects her son, Orion. Orion, who was diagnosed with the condition last year, will join his mum by scooting or riding his bike when he can. Dravet Syndrome is a rare neurological condition that includes severe epilepsy, learning disabilities, and other associated difficulties. Lewisham mum and son raise funds for rare epilepsy condition with #EveryDayinMayForDravet (Image: Dravet Syndrome UK) It affects around 1 in 15,000 live births in the UK and can result in multiple seizures day and night, with a higher risk of SUDEP (Sudden Unexpected Death in Epilepsy) compared to other forms of epilepsy. Orion experienced his first seizure at nine months old and was diagnosed with Dravet Syndrome after months of seizures, tests, and emergency hospital visits. A respiratory infection at the age of three triggered a prolonged seizure, resulting in two days in intensive care. Ms Thomas said: "Our local hospital is Lewisham hospital. "I never thought I would become so familiar with the hospital, and I have lost count of the number of times we have been there. "Orion sits on the milder end of the Dravet spectrum, but the impact on our lives has still come with many challenges. "It's so hard because you are always on high alert looking for signs of seizures. "I want to raise awareness because I feel like there's not enough regard for children with epilepsy and Dravet is much more than epilepsy. "It can feel so isolating, like you are the only one going through it." She added: "The emotional impact of Orion's diagnosis has been immense, and like so many other parents of children with complex needs, my mental health has taken such a huge hit, along with my physical health. "Stress can really take a toll on the body. "That's why I'm walking, running, cycling Every Day in May for Dravet. "To raise money and awareness of the condition, but also to take a step towards rebuilding my own physical and mental wellbeing. "Orion is so sweet and full of joy. "He has shown so much strength and resilience that it inspires me to be brave too. "He really is my hero. "I am incredibly grateful for where he is right now – but I've seen how devastating this condition can be, and how urgently more research, understanding, and family support is needed. "All donations, no matter how big or small, are greatly appreciated and make such a huge difference." The #EveryDayinMayForDravet challenge is a global initiative with hundreds of participants, many of whom are running 5k or 10k each day. It is a family-friendly challenge, with people of all ages and fitness levels taking part, including children. Galia Wilson, chair of trustees at Dravet Syndrome UK, said: "We are so grateful to Reniece and to everyone who is joining in this epic challenge, raising funds and awareness of Dravet Syndrome. "As a small charity, all donations received make a huge difference in helping us to continue our vital work supporting and empowering families, educating professionals, and funding research. "We're so thankful to them and to everyone who is taking part or digging deep for #EveryDayinMayForDravet."
7NEWS
08-05-2025
- Health
- 7NEWS
From $10k to $31.70: Massive savings for severe epilepsy sufferers after drug added to PBS
A life-changing medication for those battling a rare form of epilepsy has been added to the Pharmaceutical Benefits Scheme (PBS). Fenfluramine, an additional treatment option for those with Dravet syndrome, is a ray of hope for families dealing with an already non-responsive epilepsy. By the time this drug is recommended to people with the syndrome, they are normally on at least two other medications. 'A lifelong condition': What is Dravet syndrome? Affecting one in 15,700 people, the Dravet syndrome is characterised by seizures that are hard to treat and significant developmental delays. The syndrome typically begins when children are 6 months to 12 months old, and the mortality rate is 15 per cent to 20 per cent due to SUDEP (sudden unexpected death in epilepsy) and related accidents such as drowning and infections. And it doesn't go away when they become adults. According to Professor Piero Perucca, 'while onset begins in infancy, it remains a lifelong condition, with reduced survival'. Adults look at 'persistent seizures, disabling cognitive, behavioural, and motor symptoms', with little effect from medication. Medication can help reduce symptoms, but it is not something that can be cured. 'Constant state of fight or flight' At just four months old, Raphael Lennard had his first seizure, which proved to be long and uncontrolled. 'The early days were really quite shocking and scary,' said Raphael's father, Ben Lennard. What followed were weekly hospital visits and a constant state of fear and uncertainty, only increasing during COVID-19, when restrictions at hospital meant only one parent could be with him at a time. Seizures were 10 minutes to 20 minutes long, with multiple rounds of drugs needed to stop them. 'It was happening every week — trying to make normal life work around that was just impossible, we were just in a constant state of fight or flight,' said Lennard. There were also developmental delays and reactions to other medications that suppressed appetite. 'There's still motor delays, he doesn't want to eat so he's not food driven like other kids, we can't reward him with chocolate because he just doesn't care,' Lennard said. 'Ungodly amounts': $10k every 60 days And the medication price before the PBS? $10,000 every 60 days. This is often after trying multiple other medications, and those with Dravet syndrome can look at being on at least three anti-epileptic drugs at a time. This doesn't even take into account the cost for specialists and hospital visits. Due to the frequency of seizures and time spent in hospital, Rachael was given compassionate access to fenfluramine, without the heavy price tag. 'It has been a game changer for us, the seizures are shorter, happening only four to six months,' said Lennard. 'For other families, just having this option at the start without having to go through all the different concoctions, without having to pay ungodly amounts — it's another weapon in the arsenal against epilepsy'. Impact on carers Because Dravet syndrome is diagnosed in infancy, the burden of care and advocacy often fall on parents and guardians. Not knowing when the next seizure will come can be terrifying for child and parent and the helplessness felt watching a loved one going through one. Both of Raphael's parents have had to cut their hours to part-time work as a result. Research shows that the burden of care can lead to persistent anxiety, poor mental health and sleep quality, loss of work and financial burdens. The care burden has also resulted in 55 per cent of carers accessing a mental health or family support service for the parents or siblings of the person living with the condition. It is a condition that affects the whole family. 'Stability in the chaos' With fenfluramine on the PBS, prescriptions will now cost a maximum of $31.70, which is huge drop from the previous price of $10,000. 'It could help so many families like ours find stability in the chaos,' Lennard said. Lennard said there were also benefits for the wider community as 'we're not accessing hospitals so much, we're not going to the GP every week to get new scripts'. 'It makes life a little bit easier.'
South China Morning Post
06-05-2025
- Health
- South China Morning Post
Trial epilepsy drug reduces seizures, gives children and their families normal lives
The mother of a seven-year-old boy said 'I feel like a millionaire, like I've won the lottery', after a pioneering clinical trial transformed the life of her son, who has a severe form of epilepsy. Advertisement Albie Kelly and Freddie Truelove, both seven, are two of the children who have been given a new lease on life following the research trial at Sheffield Children's Hospital in the UK. Both boys have Dravet syndrome, a form of epilepsy which often begins before the age of one, and affects about one in every 15,000 babies born. Youngsters taking part in the trial have seen dramatic changes – moving from experiencing debilitating seizures to learning to speak, read, write independently, make new friends and take part in sport, the hospital said. A drug trial has transformed young Albie Kelly's life, his mother Lauren Kelly says. Photo: Albie's mother, Lauren Kelly, said: 'We rarely left the house out of fear that Albie might have a seizure in a place not easily accessible for an ambulance. I wouldn't travel out of the area to stay near Sheffield Children's. Since the research trial it's like I have a new child.' Advertisement Albie, who lives with his mother, father and sister in Barnsley, South Yorkshire, received the first dose of the trial drug zorevunersen, previously known as STK-001, in June 2022.
