Campaign girl 'ineligible' for muscle wasting drug
The 11-year-old is thought to be in 15% of spinal muscular atrophy patients who do not qualify. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - June 19, 2019 Category: Consumer Health News Source Type: news
Do You Consider Cost When Prescribing a Drug? Do You Consider Cost When Prescribing a Drug?
A newly approved gene therapy for children with spinal muscular atrophy will cost more than $2 million for a one-time infusion.Medscape Reader Polls (Source: Medscape Business of Medicine Headlines)
Source: Medscape Business of Medicine Headlines - June 12, 2019 Category: Pharmaceuticals Tags: Internal Medicine News Source Type: news
Medical News Today: Medical conditions that cause muscle wasting
Conditions that can cause muscle wasting include muscular dystrophy, multiple sclerosis, and spinal muscular atrophy. Treatment may include dietary changes and exercise. Learn more here. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - June 11, 2019 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news
New Gene Therapy Poised to Transform Care for Spinal Muscular Atrophy
The University of Rochester Medical Center has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy. The new treatment, which is being marketed by Novartis under the brand name Zolgensma, can be delivered within weeks of birth and clinical trials have shown that it dramatically changes the course of the disease. (Source: University of Rochester Medical Center Press Releases)
Source: University of Rochester Medical Center Press Releases - June 6, 2019 Category: Universities & Medical Training Authors: University of Rochester Medical Center Source Type: news
Monthly News Roundup - May 2019
Zolgensma: First Gene Therapy for Pediatric Spinal Muscular Atrophy (SMA)
Zolgensma (onasemnogene abeparvovec-xioi) from AveXis (a Novartis company), is now approved for children less than 2 years of age with spinal muscular atrophy (SMA).
SMA... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - May 31, 2019 Category: Pharmaceuticals Source Type: news
Newly Approved Spinal Muscular Atrophy Gene Therapy, Zolgensma ® ,...
AskBio continues to lead the path for gene therapy development and cost-effective manufacturing methods based on the contributions of its co-founder, Dr. Jude Samulski.(PRWeb May 30, 2019)Read the full story at https://www.prweb.com/releases/newly_approved_spinal_muscular_atrophy_gene_therapy_zolgensma_validates_askbio_gene_therapy_platform/prweb16344912.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - May 30, 2019 Category: Pharmaceuticals Source Type: news
FDA approves most expensive gene therapy ever — and here is its connection to Penn
The Food and Drug Administration last week approved a gene therapy product — which immediately became the country's most expensive drug at $2.125 million for the one-time treatment — that incorporates a gene delivery technology developed at the the University of Pennsylvania.
Novartis received FDA approval for Zolgensma, a one-time infusion for pediatric patients with spinal muscular atrophy (SMA) who are less than two years of age.
SMA is caused by a mutation in a gene, known as the survival… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 28, 2019 Category: Biotechnology Authors: John George Source Type: news
FDA Approves First Gene Therapy Zolgensma (Onasemnogene Abeparvovec-xioi) for Spinal Muscular Atrophy
TUESDAY, May 28, 2019 -- The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday.
Zolgensma (onasemnogene abeparvovec-xioi), an... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - May 28, 2019 Category: General Medicine Source Type: news
FDA Approves First Gene Therapy for Spinal Muscular Atrophy
TUESDAY, May 28, 2019 -- The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday.
Zolgensma (onasemnogene abeparvovec-xioi), an... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - May 28, 2019 Category: Pharmaceuticals Source Type: news
FDA Approves Gene Therapy for Spinal Muscular Atrophy
At $2 million for a single dose, Novartis ’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease. (Source: The Scientist)
Source: The Scientist - May 27, 2019 Category: Science Tags: News & Opinion Source Type: news
Girl, 15, delighted because NHS made a life-changing drug available
Abbie Bolt, 15, from Minster in Kent, suffers from spinal muscular atrophy (SMA) and has to be hooked up to a machine throughout the night to help her breathe. Pictured with mother Mel. (Source: the Mail online | Health)
Source: the Mail online | Health - May 27, 2019 Category: Consumer Health News Source Type: news
FDA Approves Zolgensma (onasemnogene abeparvovec-xioi) Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy
May 24, 2019 -- The U.S. Food and Drug Administration today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - May 26, 2019 Category: Drugs & Pharmacology Source Type: news
The $2m drug reveals medical research as a casino culture | Kenan Malik
Does Zolgensma, a revolutionary one-off treatment for spinal muscular atrophy, really need to cost so much?How much is a life worth? $2.15m? That ’s thestaggering price of a drug produced by the pharmaceutical giant Novartis that has just come on the market. Zolgensma is a one-off gene therapy treatment for spinal muscular atrophy (SMA), a rare degenerative disorder. Infants with the most severe form usually die within two years. For parents of babies born with SMA, any price is worth paying to save the child ’s life. Novartis argues that spread across a lifetime, $2.15m is “cost-effective”.It points out, too, the ...
Source: Guardian Unlimited Science - May 26, 2019 Category: Science Authors: Kenan Malik Tags: Novartis Pharmaceuticals industry NHS Medical research Science Health Society Business UK news Source Type: news
$2.1m Novartis gene therapy to become world's most expensive drug
US approves the one-time treatment for deadly spinal muscular atrophy in infantsSwiss drugmaker Novartis has received US approval for itsspinal muscular atrophy gene therapy Zolgensma – pricing the one-time treatment at a record $2.125m.The Food and Drug Administration on Friday approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest form of the inherited disease as well as those with types where debilitating symptoms may set in later.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - May 25, 2019 Category: Science Authors: Reuters Tags: Drugs Health Science Pharmaceuticals industry Business Novartis Source Type: news
This New Treatment Could Save the Lives of Babies. But It Costs $2.1 Million.
The price set by the Swiss drugmaker Novartis may be the world ’s highest for a single treatment — prompting renewed debate about how society will pay for gene-therapy breakthroughs. (Source: NYT Health)
Source: NYT Health - May 25, 2019 Category: Consumer Health News Authors: Katie Thomas Tags: Spinal Muscular Atrophy Novartis AG Drugs (Pharmaceuticals) Food and Drug Administration Health Insurance and Managed Care Genetic Engineering Gene Therapy Prices (Fares, Fees and Rates) Babies and Infants your-feed-healthcare Source Type: news
