Novartis confident of Zolgensma supply, calls $2 million price 'speculation'
Novartis is confident it has adequate production capacity for its Zolgensma gene therapy should regulators this month approve the drug for multiple forms of the genetic disease spinal muscular atrophy (SMA), the Swiss drugmaker said on Wednesday. (Source: Reuters: Health)
Source: Reuters: Health - May 8, 2019 Category: Consumer Health News Tags: healthNews Source Type: news
Roche presents data from the risdiplam pivotal FIREFISH and SUNFISH studies in spinal muscular atrophy at the 2019 AAN Annual Meeting
Roche today announced new data from the dose-finding Part 1 of the pivotal FIREFISH trial showing infants with Type 1 spinal muscular atrophy (SMA) achieved key motor milestones after one year of treatment with investigational risdiplam. (Source: Roche Media News)
Source: Roche Media News - May 7, 2019 Category: Pharmaceuticals Source Type: news
Roche presents data from the risdiplam pivotal FIREFISH and SUNFISH studies in spinal muscular atrophy at the 2019 AAN Annual Meeting
Roche today announced new data from the dose-finding Part 1 of the pivotal FIREFISH trial showing infants with Type 1 spinal muscular atrophy (SMA) achieved key motor milestones after one year of treatment with investigational risdiplam. (Source: Roche Investor Update)
Source: Roche Investor Update - May 7, 2019 Category: Pharmaceuticals Source Type: news
Act now or babies will DIE, charities warn
Matilda Jamieson, from York, has type 2 spinal muscular atrophy. She is unable to get Spinraza. However, patients in Scotland can get the drug, which is proven to work. (Source: the Mail online | Health)
Source: the Mail online | Health - May 2, 2019 Category: Consumer Health News Source Type: news
$150,000 grant will help finance research on spinal muscular atrophy
(Texas Tech University Health Sciences Center El Paso) Cure SMA recently awarded a $150,000 research grant to Laxman Gangwani, Ph.D., associate professor of Molecular and Translational Medicine, Center Cure SMA recently awarded a $150,000 research grant to Laxman Gangwani, Ph.D., associate professor of Molecular and Translational Medicine, Center of Emphasis in Neurosciences, at TTUHSC El Paso's Paul L. Foster School of Medicine. of Emphasis in Neurosciences, at TTUHSC El Paso's Paul L. Foster School of Medicine. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 1, 2019 Category: International Medicine & Public Health Source Type: news
New Roche data at the 2019 AAN Annual Meeting showcase breadth and promise of neuroscience portfolio
Roche today announced new data for its approved and investigational medicines for the treatment of neurological conditions will be presented at the 71st American Academy of Neurology (AAN) Annual Meeting from 4-10 May in Philadelphia, PA. Presentations include data from a pivotal study for risdiplam in spinal muscular atrophy (SMA), which has the potential to become the first oral treatment for this community. (Source: Roche Media News)
Source: Roche Media News - April 29, 2019 Category: Pharmaceuticals Source Type: news
New Roche data at the 2019 AAN Annual Meeting showcase breadth and promise of neuroscience portfolio
Roche today announced new data for its approved and investigational medicines for the treatment of neurological conditions will be presented at the 71st American Academy of Neurology (AAN) Annual Meeting from 4-10 May in Philadelphia, PA. Presentations include data from a pivotal study for risdiplam in spinal muscular atrophy (SMA), which has the potential to become the first oral treatment for this community. (Source: Roche Investor Update)
Source: Roche Investor Update - April 29, 2019 Category: Pharmaceuticals Source Type: news
Nusinersen Promising for Later-Onset Spinal Muscular Atrophy
WEDNESDAY, April 24, 2019 -- Limited evidence suggests that nusinersen treatment provides long-term benefits for children with later-onset spinal muscular atrophy (SMA), according to a study published online April 24 in Neurology.
Basil T. Darras,... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 24, 2019 Category: Pharmaceuticals Source Type: news
Novartis mulling $1.5 mln-$5 mln price range for SMA gene therapy
Novartis Chief Executive Vas Narasimhan said on Wednesday that the Swiss drugmaker is examining a price range for its Zolgensma gene therapy for spinal muscular atrophy (SMA) of between $1.5 million and $5 million per patient. (Source: Reuters: Health)
Source: Reuters: Health - April 24, 2019 Category: Consumer Health News Tags: healthNews Source Type: news
Second death in Novartis gene therapy trials under investigation
Novartis AG, which this week announced positive interim trial results for its experimental gene therapy for spinal muscular atrophy, on Friday said investigation is underway into whether a second trial death could be related to the treatment. (Source: Reuters: Health)
Source: Reuters: Health - April 20, 2019 Category: Consumer Health News Tags: healthNews Source Type: news
Biogen SMA drug price, Novartis estimates for its treatment far too high - U.S. group
Biogen Inc should slash the price of its spinal muscular atrophy (SMA) drug, and the $4 million to $5 million Novartis has said its experimental gene therapy for the disease is worth is excessive, an independent U.S. organization that reviews the value of medical treatments said on Wednesday. (Source: Reuters: Health)
Source: Reuters: Health - April 3, 2019 Category: Consumer Health News Tags: healthNews Source Type: news
AveXis expands world-leading gene therapy manufacturing capacity with purchase of advanced biologics therapy manufacturing campus in Longmont, Colorado
AveXis, a Novartis company, today announced it signed an agreement to purchase an advanced biologics therapy manufacturing campus in Longmont, Colorado, further expanding AveXis' production capacity as it prepares to launch Zolgensma® (onasemnogene abeparvovec-xioi[1]) an investigational gene therapy awaiting global regulatory approvals for the treatment of spinal muscular atrophy (SMA) Type 1 and for future gene therapy treatments in development. (Source: World Pharma News)
Source: World Pharma News - April 1, 2019 Category: Pharmaceuticals Tags: Featured Novartis Business and Industry Source Type: news
ICER reports on peanut allergy, RA and spinal muscular atrophy
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - March 31, 2019 Category: Drugs & Pharmacology Source Type: news
Spinal muscular atrophy: Families plead for drug to slow disease
Parents with children with a muscle wasting disease are calling for Spinraza to be offered on the NHS. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - March 6, 2019 Category: Consumer Health News Source Type: news
Six-month-old boy has just months to live after the NHS refuse to pay for a 'miracle drug'
Haris Khan, of Manchester, suffers from the genetic condition type 1 Spinal Muscular Atrophy, which makes muscles weak, leaving sufferers unable to move, swallow or even breathe. (Source: the Mail online | Health)
Source: the Mail online | Health - February 27, 2019 Category: Consumer Health News Source Type: news
