Latest news with #DuchenneMuscularDystrophy
South China Morning Post
3 days ago
- General
- South China Morning Post
‘It won't stop me': Hong Kong teen battles condition to live ‘ordinary days'
For Hayden Yung Hok-yat, a 15-year-old Hongkonger living with Duchenne muscular dystrophy (DMD), going out in a wheelchair is like tackling an obstacle course. Whenever he goes, from bustling shopping centres and restaurants to serene country parks, he must make sure there is a functional accessible toilet that is unlocked – some are closed off to prevent misuse by those without physical disabilities. The city's transport systems are largely accessible, but a smooth journey depends on an available wheelchair space, clear ramp access at the pickup and drop-off point and considerate passengers who make way for him, a rare occurrence. Unfriendly stares and intrusive questions about why he uses a wheelchair are common frustrations for the teenager. 'People like asking why I'm still in a wheelchair when my younger brother is walking, but what does it have to do with me?' Hayden said with a wry smile on his face. 'I hope people can better understand the needs of disabled people like me.'
Pink Villa
4 days ago
- Entertainment
- Pink Villa
As Ahaan Panday's Saiyaara co-star Aneet Padda takes spotlight, here's her unmissable shows and movies to watch before big screen debut
Ahaan Panday, cousin of actress Ananya Panday, is gearing up for his big-screen debut in Yash Raj Films' upcoming project Saiyaara. The film, slated for release on July 18, 2025, marks the first-ever collaboration between acclaimed director Mohit Suri and YRF. Adding to the excitement, Saiyaara will introduce a brand-new on-screen duo, Ahaan Panday and newcomer Aneet Padda. In this piece, we'll take a closer look at Aneet Padda 's journey and highlight some of her past films and series you should check out before watching Saiyaara. Aneet Padda movies and shows to watch before Saiyaara Salaam Venky Salaam Venky (2022), directed by Revathi, is a poignant Hindi drama inspired by the true story of Kolavennu Venkatesh, a young man battling Duchenne Muscular Dystrophy. The film follows Venky (Vishal Jethwa), who, despite his terminal illness, wishes to donate his organs through euthanasia, a procedure illegal in India. His mother, Sujata (Kajol), supports his decision, leading to a legal battle that challenges societal norms and laws. Aneet Padda portrays Nandini, Venky's visually impaired childhood friend and love interest, whose bond with him adds emotional depth to the narrative. The ensemble cast includes Rajeev Khandelwal as Dr. Shekhar Tripathi, Rahul Bose as lawyer Parvez Alam, Priyamani as public prosecutor Nanda Kumar, Aahana Kumra as journalist Sanjana, and Prakash Raj as Judge Anupam Bhatnagar. Salaam Venky is a heartfelt exploration of life, love, and the right to die with dignity. Big Girls Don't Cry In the Amazon Prime Video series Big Girls Don't Cry, Aneet Padda portrays Roohi Ahuja, a vibrant and resourceful student at Vandana Valley Girls' School. Roohi often uses humor and charm to mask the emotional turmoil stemming from her parents' troubled marriage. Her close friendship with Jayshree "JC" Chhetri (played by Tenzin Lhakyila) is central to the narrative, especially as both girls navigate a complex love triangle involving a student from a neighboring boys' school. The series delves into the lives of several students, including Kavya Yadav (Vidushi), a scholarship student striving to fit in; Leah Ludo Joseph (Avantika Vandanapu), a basketball captain grappling with her sexual identity. Noor Hassan (Afrah Sayed), an ambitious student facing cultural challenges; Anandita Pluggy Rawat (Dalai), an aspiring writer; and Dia Malik (Akshita Sood), a rebellious poet. Set against the backdrop of an elite boarding school, the show explores themes of friendship, identity, and adolescence. Apart from that, Aneet Padda was also seen in several advertisements. Very few people know that Aneet is also a singer and a songwriter, and she lent her voice to a few songs in Big Girls Don't Cry. Saiyaara teaser out The recently released teaser offers a glimpse into an intense love story, showcasing the chemistry between Ahaan Panday and Aneet Padda. Set against a backdrop of passion and heartbreak, the narrative follows the journey of two young lovers navigating the complexities of their relationship. The teaser features evocative visuals, including a music festival sequence with over 1,000 background artists, highlighting Ahaan's character as a passionate performer. Fan reactions to Saiyaara teaser Fans quickly flooded the comments section, sharing their excitement. One user said, "Woww. Going to be interesting. First time collab of Mohit Suri with YRF. Sure shot superhit banger album of this year." Another comment stated, "Combo of Aashiqui 2 + Ek Villain + Malang + Ek Villain Returns = Saiyaara." Someone else added, "Can we officially admit this, Bollywood bringing back its old music back into the game?" Another enthusiastic fan wrote, "Mohit Suri, if you're reading this, never stop making films man. Aap jaisa Music bohat kam directors samajhte hain, eagerly waiting for the full album!" Produced by Akshaye Widhani and directed by Mohit Suri, the film marks the Bollywood debut of Ahaan Panday, cousin of actress Ananya Panday, and features Aneet Padda as the female lead. Saiyaara is all set to hit theatres on July 18, 2025.
Reuters
5 days ago
- Business
- Reuters
PepGen to discontinue development of experimental Duchenne therapy; shares drop
May 28 (Reuters) - Drug developer PepGen (PEPG.O), opens new tab said on Wednesday it will discontinue development of its experimental therapy for Duchenne muscular dystrophy (DMD) after it failed to boost production of a protein in a mid-stage study. Shares of the company were down 9.5% at $1.43 in extended trading. The therapy PGN-EDO51 did not achieve target levels of dystrophin production. Dystrophin is a protein whose absence leads to muscle weakening in patients with DMD. DMD affects an estimated one in 3,500 male births worldwide, according to the National Organization for Rare Disorders. It weakens skeletal and heart muscles, deteriorating them quickly over time, and patients often die by the time they are 25. The company said it intends to wind down all DMD-related research and development activities, shifting its focus to a therapy that is being developed for a type of myotonic dystrophy, another muscle wasting disorder. PepGen expects to report data from an early-stage study in myotonic dystrophy in the second half of the year, and a mid-stage study in the first quarter of 2026.
BBC News
6 days ago
- General
- BBC News
Duchenne Muscular Dystrophy: Penrith family's Givinostat fight 'almost over'
A family say it is outrageous they have had to fight for access to a free drug that slows the progression of a muscle-wasting was conditionally approved in December to help slow the progression of Duchenne Muscular Dystrophy (DMD), but was only available in Leicestershire and Swansea due to local NHS budget Newcastle Hospitals Trust, which treats DMD patients across the North East and Cumbria, said it was "finalising arrangements" to make it available for those who meet strict eligibility family of 10-year-old Macaulay from Penrith, Cumbria, welcomed the news but have spent months of campaigning for access. Usually affecting boys in early childhood, DMD is one of the most common and severe forms of muscular dystrophy, which gradually causes the muscles to weaken, leading to an increasing level of drug was initially offered for free under an early access programme, while it awaited full NHS health bosses had to fund the staffing for treating patients, blood tests and collecting mum Rebecca has been campaigning, getting political support and writing to Newcastle Hospitals Trust to get access in the region. Rebecca said the drug would give her son a "longer, more independent life".Macaulay, who loves sports, said he has had to cut back as the genetic disease progresses."I don't think I can run as fast as I used to because I'm on steroids," he dad Ben said the continued wait was frustrating."It's confidence, it's the ability to keep up with his peers for as long as possible, the ability to walk for him is really important," he said. In a statement, the trust said: "We are pleased to confirm that we will be able to make Givinostat available through an early access programme to eligible patients in our clinics very soon."Its clinical team would discuss the drug with patients who are potentially eligible at their next review appointments, the trust Rebecca criticised the lack of timeline commitment, adding: "Yes, 'It will come soon', but what does soon mean?"Co-founder of charity Duchenne UK, Emily Reuben, said it was "time critical" as the drug was "only available for boys who can walk"."Some boys who were eligible for this drug six months ago are no longer eligible, because they've stopped walking," she said. Follow BBC North East on X and Facebook and BBC Cumbria on X and Facebook and both on Nextdoor and Instagram.
Daily Record
25-05-2025
- Health
- Daily Record
Scots kids with muscular dystrophy get new life-extending treatment
Families began receiving calls from doctors last week to arrange treatments for new Givinostat drug after a campaign by the Sunday Mail. Children with a rare type of muscular dystrophy received calls from doctors last week to begin treatment with a new life extending drug after a Sunday Mail campaign. Givinostat slows the progress of the condition but Scottish health boards had until now failed to provide it despite a free early access programme. However Health Secretary Neil Gray has now intervened and families - who had threatened legal action through their lawyer Aamer Anwar - have now been contacted to arrange first doses. There are only thought to be around 30 boys in Scotland suitable for the drug which can give extra life outside of a wheelchair to young patients. Tragically average life expectancy is just 26 years for Duchenne Muscular Dystrophy (DMD). Mhairi Harvey, whose 7-year-old son Michael has DMD and will now have access to givinostat, said: 'It's hard to put into words how we feel about having access to givinostat. 'A rollercoaster it has been, but not a fun one that I'd like to repeat. The support we've received has been truly staggering, none more so than from Aamer Anwar and his team. "We are in no doubt, we wouldn't have got this across the line at this stage for all eligible boys in Scotland without them. The Sunday Mail have been magnificent in keeping us in the public's hearts and minds. 'The kindness and empathy shown to us by so many has kept us buoyant when we thought we were sinking and we'll be forever grateful.' Nicola Rankin, whose son Michael, 12, will also get the drug added: 'I cannot begin to express the depth of my gratitude for Aamer and his team. 'After an exhausting fight to secure the treatment, Michael will now start it in the summer. It was their unwavering support, determination, and expertise that finally made it possible. "We would not have gotten this far without their help.' Karla Millar's son Sam, 8, is also looking forward to beginning his treatment. She said: 'As a family, we are over the moon to have finally been told that Sam will receive his medication over the summer. 'It is fantastic news for all families who have received their phone calls.' In a letter to Gray, Anwar said: 'Progress has been made at pace within the Glasgow and Lothians region, with parents now receiving phone calls to confirm upcoming appointments to begin administering Givinostat to their children. 'The families of these boys wish to express their sincere gratitude and appreciation for your efforts in advancing this matter. 'The steps you have taken to enforce the administration of Givinostat has been critical in shifting bureaucracy to act, offering the children an opportunity to experience a better quality of life.' However Anwar also warned that a small number of children in the Grampian area were still not being given access to the drug. Join the Daily Record WhatsApp community! Get the latest news sent straight to your messages by joining our WhatsApp community today. You'll receive daily updates on breaking news as well as the top headlines across Scotland. No one will be able to see who is signed up and no one can send messages except the Daily Record team. All you have to do is click here if you're on mobile, select 'Join Community' and you're in! If you're on a desktop, simply scan the QR code above with your phone and click 'Join Community'. We also treat our community members to special offers, promotions, and adverts from us and our partners. If you don't like our community, you can check out any time you like. To leave our community click on the name at the top of your screen and choose 'exit group'. If you're curious, you can read our Privacy Notice. And one parent in the NHS Grampian area said: 'To my knowledge, there are four boys in Grampian who should be eligible for the early access programme including my son. 'I am incandescent. Not only has the time scale been set back a further two months to October but the caveat of 'providing there are no unexpected resource limitations in this timeline' is wholly unacceptable. 'I can not express the level of upset this has caused us as parents.' Emily Reuben and Alex Johnson, founders of Duchenne UK, said: 'There's something worse than having no treatment - and that's having a treatment, available to the NHS for free, but no one can work out to administer it.'
