Latest news with #Spinraza
Irish Independent
6 hours ago
- Health
- Irish Independent
Wonder drug changed the life of a 15 year old girl now sitting the Junior Certificate, exams her parents thought she would never get to do
Robeen native Grace O'Malley (15) and her family look back on the fight for Spinraza, a ground-breaking drug which has improved the lives of those suffering Spinal Muscular Atrophy (SMA) Today at 08:27 Every morning, Grace O'Malley (15) is hoisted out of her bed in Robeen in south Mayo. It's always been this way. She has never, and will never walk because of a condition called Spinal Muscular Atrophy (SMA).
Irish Independent
11 hours ago
- Health
- Irish Independent
Wonder drug changed the life of a 15 year old girl now sitting the Junior Certificate, exams her parents thought she never get to do
Robeen native Grace O'Malley (15) and her family look back on the fight for Spinraza, a ground-breaking drug which has improved the lives of those suffering Spinal Muscular Atrophy (SMA) Every morning, Grace O'Malley (15) is hoisted out of her bed in Robeen in south Mayo. It's always been this way. She has never, and will never walk because of a condition called Spinal Muscular Atrophy (SMA).
Daily Record
22-05-2025
- Entertainment
- Daily Record
Scots schoolboy told he wouldn't live past first birthday granted sweet Spider-Man wish
A Scots schoolboy who was told he wouldn't live to see his first birthday but continued to overcome the odds has received a Disney Wish to met his superhero idol Spider-Man. Max Farmer, from Perth, was born healthy but then diagnosed with spinal muscular atrophy (SMA) type 1 – a rare and genetic condition that causes progressive muscle wasting - when he was just nine weeks old. Doctors told his heartbroken parents he wouldn't live beyond 12 months. However, Max soon became the first child in Scotland to receive a groundbreaking new treatment. Last week, the now eight-year-old's dream came true when he had a private meet-and-greet with Spider-Man at a special Make-A-Wish UK event in partnership with Disney. Mum Elaine Donoghue, 48, told the Record: "It was absolutely magical and Max was so captivated by the characters he saw. It was such an inspirational and magical experience for our whole family. "We had no idea he'd get to meet Spider-Man one-on-one. He showed him his Spidey moves and even introduced him to Strictly's Amy Dowden, it was unbelievable." The event at Hoar Cross Hall Hotel in Burton-on-Trent was a specially designed experience for children with life-limiting conditions. It was made possible through a referral from CHAS (Children's Hospices Across Scotland), where Max receives respite care through Rachel House in Kinross. At nine weeks old Max stopped moving which led to the diagnosis of the condition that not only affects muscles in the arms and legs but also those needed for breathing and swallowing. It was then Max was offered a place on a new drug trial just weeks later, becoming the first child in Scotland to get the Nusinersen (Spinraza) treatment through an expanded access programme. Elaine said: "We were told he wouldn't make it to his first birthday. It was heartbreaking. "He went through 21 lumbar punctures in five years, but it gave him a chance at life." Now, Max is on a daily oral drug called Risdiplam, which means he no longer needs hospital stays or general anaesthetic to receive treatment. Despite needing 24/7 care, Max is full of life. He goes to school every day, uses a power chair, and receives constant support from mum Elaine, dad Stewart, 47, and big sister Beth, 17. Elaine said: "His day starts at 6.30am with medication and feeding, but he never complains. He's got a huge team around him, and he just takes it all in his stride. "There are no words to describe what the experience done for us as a family. We wouldn't even be able to take him to Euro Disney in Paris, due to the equipment and meds, it would be unachievable. 'But this event was fully accessible. There were profiling beds, hoists, proper changing places – everything we needed. They covered everything. "It was a dream come true for him to meet Spider-Man and we made memories which will last a lifetime." Max, who is under the care of nearly every major hospital in Scotland, continues to defy expectations. "He's just incredible. He's happy, he's funny, he loves school - and he's living his best life, in the way he can. We are exceptionally lucky to be able to call ourselves his parents." 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'.
Yahoo
13-05-2025
- Business
- Yahoo
DRI Healthcare Trust Announces Voting Results of AGM
TORONTO, May 13, 2025 /CNW/ - DRI Healthcare Trust (TSX: (TSX: DHT.U) ("DRI" or "the Trust") is pleased to announce results of voting at its annual general meeting ("AGM") of unitholders held on May 13, 2025. All of the nominees for election as trustees of the Trust referred to in its management information circular dated March 31, 2025 were elected to serve as trustees for the ensuing year, and Deloitte LLP was reappointed as auditors of the Trust. Election of Trustees Voting results for the election of each trustee were as follows: Trustee Votes "for" as a percentage of votes cast for or withheldfor the trustee Votes "withheld" as a percentage of votes cast for or withheld for the trustee Gary Collins 88.19 % 11.81 % Ali Hedayat 90.22 % 9.78 % Kevin Layden 90.13 % 9.87 % Athana Mentzelopoulos 99.75 % 0.25 % Paul Mussenden 88.65 % 11.35 % Poonam Puri 89.58 % 10.42 % Tamara Vrooman 88.26 % 11.74 % Appointment of Auditors Unitholders also voted in favour of reappointing Deloitte LLP as auditors of the Trust and authorizing the board of trustees to fix their remuneration (95.75% in favour). About DRI Healthcare Trust The Trust is managed by DRI Capital Inc., a pioneer in global pharmaceutical royalty monetization. Since its initial public offering in 2021, the Trust has deployed more than $1.0 billion, acquiring more than 25 royalties on 20-plus drugs, including Eylea, Orserdu, Omidria, Spinraza, Stelara, Vonjo, Zejula and Zytiga. The Trust's units are listed and trade on the Toronto Stock Exchange in Canadian dollars under the symbol " and in U.S. dollars under the symbol "DHT.U". To learn more, visit or follow us on LinkedIn. SOURCE DRI Healthcare Trust View original content to download multimedia: Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
01-05-2025
- Business
- Yahoo
Biogen's results beat estimates on strength in rare disease drugs
(Reuters) -Biogen beat first-quarter profit and revenue expectations on Thursday, as strong demand for its rare disease drugs helped offset declining sales of its multiple sclerosis medicines. The drugmaker has focused on deals, cost-cutting measures and newer drugs to address investor pressure for growth as demand falls for its once top-selling multiple sclerosis medicines in the face of stiff competition in a crowded treatment market. The "relatively calm quarter" for Biogen showed positive signs of improvement, said BMO Capital Markets analysts. Biogen has been doubling down on its Alzheimer's drug Leqembi, but it has failed to live up to lofty expectations due to concerns over cost, efficacy and side effects. It sells for $26,500 annually in the U.S. The drugmaker said it does not expect any material impact this year from the Trump administration's sweeping tariffs implemented so far to boost domestic manufacturing of goods. Biogen said about 75% of its U.S. revenue last year came from products manufactured in the country. Drugs primarily manufactured in the U.S. include Leqembi and spinal muscular atrophy treatment Spinraza. The company expects 2025 profit per share of $14.50 to $15.50, compared with its previous forecast of $15.25 to $16.25. The forecast cut reflects a $165 million charge related to its licensing deal with Stoke Therapeutics earlier this year. It reported quarterly adjusted profit of $3.02 per share, beating analysts' expectations of $2.52 per share, according to estimates compiled by LSEG. U.S. sales of Leqembi, which the company sells with Japan's Eisai, were $52 million for the first quarter. The Wall Street consensus estimate was at $51 million, according to brokerage Jefferies. First-quarter sales of its multiple sclerosis drugs fell 11% to $953 million, while sales of rare disease drugs, including Skyclarys and Spinraza, jumped 33% to $563 million. Revenue rose 6% to $2.43 billion, and beat expectations of $2.23 billion. Sign in to access your portfolio
