The program aims to expand access to a potentially life-saving drug that has only been approved in the US. Please see the Full Prescribing Information. The program is for patients in countries where the medicine, called Zolgensma, is not yet approved for the rare genetic disorder, which can lead to death and profound physical disabilities. Novartis said the program will open for submission on Jan. 2 and the first allocation of drugs would begin in February.
Copyright © 2020 GenomeWeb, a business unit of Crain Communications. Learn about the clinical study results in children with SMA. “In this case, the original diagnosis and that association hasn’t held true.”. Lucy Frost, whose eight-year-old son George has the disease and who is a trustee of TreatSMA in the UK, said the fortnightly draw would be very hard on families. He said no account would be taken of the severity of the child’s disease or the speed at which it was progressing. The Novartis lottery is in full swing. Use of this site is subject to our terms of use, privacy policy, advertisement policy. What causes SMA? “We are yet to be convinced that a health lottery is an appropriate way of meeting the unmet medical needs in this severe disease,” the British advocacy group TreatSMA said in a statement. Desperate parents and doctors have registered children for this Novartis publicity stunt and find it hard to compete in the “lottery for life. It will have a huge emotional impact,” she said. © 2020 Guardian News & Media Limited or its affiliated companies. Check out more about our bureau/team here, • Email: info@medicaldialogues.in• Phone: 011 - 4372 0751. The company's AveXis unit, which developed the drug- Zolgensma, will give out 50 doses of the treatment through June for babies under 2 years old, Novartis said, with up to 100 total doses to be distributed through 2020. Therapy Lottery Dec 23, 2019 The plan to hold a lottery to give away 100 free doses of the gene therapy Zolgensma has been met with consternation, the Guardian reports . ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). Criticism of Novartis/Avexis’s controversial lottery style managed access program for Zolgensma has come largely from Europe, while other countries in Latin America and Asia have been … According to Patgiri, “the best outcome for all patients would be if everybody could get the treatment. Do Not Sell My Personal Information. © 2020 Minerva Medical Treatment Pvt Ltd, Medical Dialogues Bureau consists of a team of passionate medical/scientific writers, led by doctors and healthcare researchers. Privacy Policy. The importance of newborn screening for SMA. In Cell this week: SARS-CoV-2 evolution and spread in North Carolina, protein and other features that distinguish mild and severe COVID-19, and more. It is designed to add a functional copy of a gene that is missing in babies born with SMA. To improve access to SMA treatment, Novartis announced a plan to give away 100 doses of Zolgensma at no cost in 2020 through a lottery program.