Thanks To “Miracle” Drug Zolgensma £1.8million, Little Boy Has A Chance Of A ‘Normal Life’
Most children with type 1 sᴘɪɴᴀʟ ᴍᴜsᴄᴜʟᴀʀ ᴀᴛʀᴏᴘʜʏ ᴀʀᴇ ᴇxᴘᴇᴄᴛᴇᴅ to only ʟɪᴠᴇ a few years, but thanks to “ᴍɪʀᴀᴄʟᴇ” ᴅʀᴜɢ Zᴏʟɢᴇɴsᴍᴀ, little Marley Powell has a chance of a ‘normal life’A baby boy has been given a chance of a “normal life” after receiving ᴀ ᴅᴏsᴇ of the “ᴍᴏsᴛ ᴇxᴘᴇɴsɪᴠᴇ ᴅʀᴜɢ in the ᴡᴏʀʟᴅ”. Marley Powell, 16 months, was ᴅɪᴀɢɴᴏsᴇᴅ with Type 1 sᴘɪɴᴀʟ ᴍᴜsᴄᴜʟᴀʀ ᴀᴛʀᴏᴘʜʏ (SMA), a ɢᴇɴᴇᴛɪᴄ ᴄᴏɴᴅɪᴛɪᴏɴ ᴡʜɪᴄʜ ᴄᴀᴜsᴇs ᴀ ʀᴀɴɢᴇ of ʟɪFᴇ-ʟɪᴍɪᴛɪɴɢ ᴘʀᴏʙʟᴇᴍs. Most children with the ᴄᴏɴᴅɪᴛɪᴏɴ are expected to only ʟɪᴠᴇ a few years, but doctors have called the ᴅʀᴜɢ Zᴏʟɢᴇɴsᴍᴀ, which was licensed in March, a “miracle”.
It was made available on the NHS after a deal was struck with manufacturers Novartis Gᴇɴᴇ Therapies, and a one ᴏFF ᴅᴏsᴇ ᴄᴏsᴛs £1.79 million. Parents Rosie-Mae Walton and Wes Powell, from Driffield, Hull, told Hullʟɪᴠᴇ they faced a “rollercoaster” waiting to find out if he would be one of the first 30 children in the country to receive the ᴍᴇᴅɪᴄᴀᴛɪᴏɴ
Speaking to Hullʟɪᴠᴇ after the ᴛʀᴇᴀᴛᴍᴇɴt, Rosie-Mae said: “We feel amazing that Marley has had the ᴛʀᴇᴀᴛᴍᴇɴt. Marley is currently doing ᴀᴍᴀᴢɪɴɢʟʏ ᴀFᴛᴇʀ ʜɪs ᴛʀᴇᴀᴛᴍᴇɴt. We were unsure at first if he was going to be eligible for Zᴏʟɢᴇɴsᴍᴀ so when we found out he was and the ᴀɴᴛɪʙᴏᴅʏ ᴛᴇsᴛ came back ɴᴇɢᴀᴛɪᴠᴇ we just cried and cried with happiness all day, because we finally knew he could ʜᴀᴠᴇ ɪᴛThis ᴅʀᴜɢ is the ᴍᴏsᴛ ᴇxᴘᴇɴsɪᴠᴇ ᴅʀᴜɢ in the ᴡᴏʀʟᴅ and he deserves it.”Rosie-Mae added: “Zᴏʟɢᴇɴsᴍᴀ could give Marley the possibility to sit unaided and even walk. It’s such a surreal feeling knowing he’s ʜᴀᴅ ɪᴛ because it’s just a one-time ᴛʀᴇᴀᴛᴍᴇɴt and you just connect it to a drip over one hour and it’s done. It was just the happiest moment ever, just to know that he’s actually getting it, after all the time waiting and how much we’ve been through and how much Marley’s been through. Then you think that he’s had this ᴍᴏsᴛ ᴇxᴘᴇɴsɪᴠᴇ ᴅʀᴜɢ in the ᴡᴏʀʟᴅ. It’s just ᴄʀᴀᴢʏ. It’s the best thing ever.”
The couple said money raised for Marley will be used for continuing ᴛʀᴇᴀᴛᴍᴇɴt, including ᴘʀɪᴠᴀᴛᴇ ᴘʜʏsɪᴏᴛʜᴇʀᴀᴘʏ ᴀɴᴅ ᴇǫᴜɪᴘᴍᴇɴᴛ.
Rosie-Mae said: “As much as he’s got his ᴛʀᴇᴀᴛᴍᴇɴt, it’s not just going to be an easy ride. So, we just thank every single person that’s helped out for that.”
Tony Hart, ᴄᴏɴsᴜʟᴛᴀɴᴛ ᴘᴀᴇᴅɪᴀᴛʀɪᴄ ɴᴇᴜʀᴏʟᴏɢɪsᴛ at Sheffield Children’s Hospital, said: “Zᴏʟɢᴇɴsᴍᴀ is a breakthrough because it really is a ᴍɪʀᴀᴄʟᴇ ᴅʀᴜɢ. Without Zᴏʟɢᴇɴsᴍᴀ, most children would have ᴅɪᴇᴅ.”
She said Marley appears to be doing really well since Tuesday’s ᴛʀᴇᴀᴛᴍᴇɴt. “It feels so surreal, like it hasn’t hit home yet that he’s actually ʜᴀᴅ ɪᴛ – that a drip is just so expensive and has worked wonders. It’s just ᴄʀᴀᴢʏ considering all we’ve been through and the amount of scares you get put through thinking that your baby won’t reach the age of two or a second birthday.”
Mr Hart said more than 30 children have now received Zᴏʟɢᴇɴsᴍᴀ in the four centres in England and Wales where is it being given – Sheffield, London, Manchester and Bristol.
Asked about handling the “ᴍᴏsᴛ ᴇxᴘᴇɴsɪᴠᴇ ᴅʀᴜɢ in the ᴡᴏʀʟᴅ”, the consultant said he leaves that to the nurses, adding: “One of the great things about the NHS is that decisions about costs are taken out of our hands and our ᴘʀɪᴏʀɪᴛʏ is giving as many children who need it the ᴅʀᴜɢ when we can, and as fast as we can. Even though Marley has had the ᴛʀᴇᴀᴛᴍᴇɴt
What is Sᴘɪɴᴀʟ ᴍᴜsᴄᴜʟᴀʀ ᴀᴛʀᴏᴘʜʏ (SMA)?
Sᴘɪɴᴀʟ ᴍᴜsᴄᴜʟᴀʀ ᴀᴛʀᴏᴘʜʏ (SMA) is a ɢᴇɴᴇᴛɪᴄ (inherited) ɴᴇᴜʀᴏᴍᴜsᴄᴜʟᴀʀ ᴅɪsᴇᴀsᴇ that causes ᴍᴜsᴄʟᴇ to become weak and waste away. People with SMA lose a specific type of nerve ᴄᴇʟʟ ɪɴ ᴛʜᴇ sᴘɪɴᴀʟ cord (called motor neurons) that control muscle movement. Without these motor neurons, ᴍᴜsᴄʟᴇ don’t receive nerve signals that make ᴍᴜsᴄʟᴇ move. The word atrophy is a medical term that means smaller. With SMA, certain ᴍᴜsᴄʟᴇ become smaller and weaker due to lack of use.
How common is sᴘɪɴᴀʟ ᴍᴜsᴄᴜʟᴀʀ ᴀᴛʀᴏᴘʜʏ?
Approximately 10,000 to 25,000 children and adults are living with SMA in the United States. It’s a rare ᴅɪsᴇᴀsᴇ ᴛʜᴀᴛ ᴀFFᴇᴄᴛs one out of 6,000 to 10,000 children.