Latest news with #Duchennemusculardystrophy
Daily Record
04-07-2025
- Health
- Daily Record
Dad of tragic Scots boy in government help call after selling home amid treatment nightmare
Jamie Tierney, whose son is terminally ill, has sold the family home. The dad of a tragic Scots boy has called for more help for families with terminally ill children - after selling their home while shelling out thousands of pounds on his treatment. Devoted dad Jamie Tierney believes there should be a body set up to help families navigate the difficulties a terminal diagnosis brings, from accessing benefits to help with housing and other issues. The 33-year-old, of Dunfermline, Fife, said he was forced to make his family homeless as it was the best option due to the difficult circumstances they find themselves in. They can put money from the sale of their home towards treatment for their six-year-old son, also Jamie, who suffers from rare muscle wasting disease Duchenne muscular dystrophy (DMD). Jamie senior said the youngster's condition meant the family home had become unsuitable for him, and they are now staying with different family members as they wait to hear if they will be given a council house. They receive support from the Muscular Dystrophy UK charity, and the NHS, as well as carers' allowance, but have to deal with each organisation individually, and Jamie believes a streamlined service would make it easier for families. Jamie's call for change comes after the family were denied the chance for Jamie junior to get a wonder medication which is provided free to the NHS. The family has headed abroad on a number of occasions for Jamie to receive treatment. Givinostat, a groundbreaking new life-extending treatment which slows the progress of the condition, has been cleared for use in the UK and is being used in England. 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. But Scottish health boards have not given the drug to any patients through an early access programme, despite patients south of the border benefitting from it. Jamie, 33, said: "We sold our family home for many reasons — it was a townhouse with too many stairs, completely unsuitable for Jamie. We needed to find a way to give him independence. " Jamie's needs are increasing year by year. Our street is on a hill, and watching the kids play there breaks my heart — knowing Jamie can't join in this summer. "We need to keep money for future treatments and medical needs for Jamie. I think there should be supportive pathways for families who are in this situation and to aid them with options and help as currently we are jumping through hoops. "We were told we were the last family accepted on a clinical trial in the U.S. We were petrified but ready to move for Jamie. It fell through — we were devastated." The family have headed to the United States and Mexico for treatments for Jamie, paid for with the help of fundraising from raffles and GoFundMe pages. The incurable muscle-wasting disease, which gradually weakens all muscles in the body, affects one in 5,000 boys and sees sufferers having a life expectancy of around mid-20s. Jamie added: "As of this Friday, we're technically homeless. We can't buy again — my wife had to give up work to care for Jamie, and I'm in the early stages of building a new business that just about keeps us afloat. "Housing are trying to help, but we're told we just meet the criteria of 'homeless'. Yes, we could rent privately — but a suitable ground-floor property would cost double our old mortgage. "The system isn't built for families in extreme, complex situations and when you don't fit the system the system forgets you. How are families supposed to survive like this? I think there's certainly got to be some sort of help. "If we weren't in this situation my wife and I would both be working. She is no longer working and the stress and worry on your child's health affects your work. I'm in a first year start up struggling to support us. "If we had to go private rent it would financially cripple us. We are self funding Jamie's treatments with fundraising but fundraising has become harder and harder for us. We just feel sorry for other families in this situation. "There's families that are being affected every day. I wouldn't wish this on anyone. We would live on the streets for Jamie to be healthy, but the system doesn't seem to consider us." You can donate to their GoFundMe page here. The Scottish Government said: "We have every sympathy with the Tierney family and the hardships they're facing. We want disabled people and carers to get the support they are entitled to, making sure the application process is as straightforward as it can be. " Child Disability Payment is designed to mitigate some of the additional costs of caring for a disabled child or young person and we have a fast-track application route for people who have a terminal condition. "We have also transformed financial support for unpaid carers in Scotland, in recognition of the impact caring can have on a family's finances. "Local authorities will be provided with £15 billion this financial year for a range of services, including housing options services – an advice process councils use when someone approaches them with a housing problem."
Business Wire
16-05-2025
- Business
- Business Wire
CAPR Investors Have Opportunity to Join Capricor Therapeutics, Inc. Fraud Investigation with the Schall Law Firm
LOS ANGELES--(BUSINESS WIRE)-- The Schall Law Firm, a national shareholder rights litigation firm, announces that it is investigating claims on behalf of investors of Capricor Therapeutics, Inc. ('Capricor' or 'the Company') (NASDAQ: CAPR) for violations of the securities laws. The investigation focuses on whether the Company issued false and/or misleading statements and/or failed to disclose information pertinent to investors. Capricor announced on May 5, 2025, that following "the completion of a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for the Company's Biologics License Application (BLA) seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy", the agency "confirmed its intent to hold an advisory committee meeting" regarding the Company's BLA. If you are a shareholder who suffered a loss, click here to participate. We also encourage you to contact Brian Schall of the Schall Law Firm, 2049 Century Park East, Suite 2460, Los Angeles, CA 90067, at 310-301-3335, to discuss your rights free of charge. You can also reach us through the firm's website at or by email at bschall@ The Schall Law Firm represents investors around the world and specializes in securities class action lawsuits and shareholder rights litigation. This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.
Fox News
22-02-2025
- Health
- Fox News
I'm dying of a fatal disease. Bureaucrats shouldn't decide how doctors can try to save me
At age 6, I was diagnosed with a fatal disease. At age 11, I lost my ability to walk. Today, I'm 23 and am fighting for my life — and for all of those who are desperate for a cure. Tragically, the federal government is standing in the way. I'm not alone; tens of millions of Americans suffer from rare diseases. And while I believe we have entered a "Golden Age" of personalized medicine tailored around a patient's DNA, regulatory roadblocks often force us to wait years to access treatments that could help. We need to break through this logjam so patients can access cutting-edge treatments today. My disease is Duchenne muscular dystrophy (DMD) — a rare, genetic disorder that causes my muscles to waste away. Patients lose their ability to walk around the ages 9 to 12 and become completely dependent on a power-wheelchair. Then they noticeably lose function of their arms in their teens, and eventually lose their life in their mid-20s as the muscles responsible for breathing are affected, as well as the heart muscle. I decided early on not to let this disease define me — or stop me. At 15, I founded a nonprofit organization called Destroy Duchenne. I went on to become a bestselling author, public speaker, Hollywood writer and producer, biotech consultant to five companies, investor, and recognized Key Opinion Leader (KOL) in DMD. Yet I still know the brutality and pain this disease causes. My younger brother, who is 17, also has DMD. So did my other younger brother, who passed away from the disease at age 14. Duchenne affects not just the person with the disease, but everyone around them, as it steals the person away from those who love them. Fortunately, I'm optimistic about innovation in the medical field. As someone who works in the biotech industry, I know of countless advances currently in the works that are going to change the way we deal with diseases. With the rise of AI, even more progress can be made faster and cheaper, and drugs can be even safer from their inception. What is critical with rare genetic diseases is time. It's nearly always better to intervene earlier, as it is easier to preserve existing function and overcome some of the effects of the disease. For example, younger patients with Duchenne tend to have more muscle. They also tend to have less fibrosis (scar tissue) and fat infiltration — two biological effects of the disease as time progresses, which further reduce mobility. Tragically, some patients are at the end of their life, where days can make the difference between living or dying. On average, clinical trials for new treatments take around 10 years to complete. By then, a patient's disease may have progressed beyond the point where they can be meaningfully treated — or they may have even passed away. We need legislation like the "Right to Try for Individualized Treatments." It's an innovative reform that's now law in six states that puts the power into the hands of the doctor and patient to make these critical decisions about when to try investigational new drugs that are customized based on genetics. Right now, I can be willing to try a new treatment, the manufacturer of the treatment can be willing to allow me to try it, and a doctor can be willing to administer it — yet Uncle Sam is not willing and stops me from receiving a lifesaving treatment. This is completely backwards. After all, doctors know their patients better than a far-off bureaucrat who doesn't even know the patient exists. Why should I have to beg the federal government for permission to try to save my own life? The reality for me is that I'm going to die, likely before I reach middle-age. The average lifespan for people with Duchenne is 25. But it doesn't have to be that way.
