New two-year data show Roche ’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA)
Basel, 16 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new exploratory 2-year longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi ™ (risdiplam) in people aged 2-25 years with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). The study suggests that gains in motor function observed with Evrysdi treatment at month 12 continued to improve or were maintained at month 24 across primary and secondary endpoint measures. Based on the natural history of the disease, people with Types 2 and 3 SMA who remain untreated decline in motor function over time. These ...
Source: Roche Investor Update - March 16, 2021 Category: Pharmaceuticals Source Type: news
Risdiplam Promising for Spinal Muscular Atrophy Risdiplam Promising for Spinal Muscular Atrophy
Infants with type 1 spinal muscular atrophy showed increased expression of functional survival motor neuron protein after 1 year of treatment in new FIREFISH findings.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - March 10, 2021 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
Spinal muscular atrophy: Gene therapy approved by NHS
Drug treatment for fatal genetic disorder will be the most expensive approved in the UK. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - March 8, 2021 Category: Consumer Health News Source Type: news
NHS to use world's most expensive drug to treat spinal muscular atrophy
Zolgensma, which costs £1.79m for one-off treatment, will be available in England this year for the first timeThe world ’s most expensive drug, which treats babies and young children with a rare and often fatal degenerative disorder, will be available this year for the first time on the NHS in England.Zolgensma, which costs £1.79m per dose, halts the progression ofspinal muscular atrophy (SMA), which involves loss of movement, muscle weakness and paralysis, and is the leading genetic cause of death in infants.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - March 8, 2021 Category: Science Authors: Phoebe Weston Tags: NHS Drugs Medical research Health Children Pharmaceuticals industry Society UK news England Source Type: news
First Oral SMA Treatment Gets EMA Panel Thumbs Up First Oral SMA Treatment Gets EMA Panel Thumbs Up
The CHMP gives positive opinion on risdiplam as the first oral treatment for adults and children at least 2 months of age who have certain types of spinal muscular atrophy.International Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - March 1, 2021 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news
NICE recommends Zolgensma for type 1 spinal muscular atrophy
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - March 1, 2021 Category: Drugs & Pharmacology Source Type: news
Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older
Basel, 26 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi ™ (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease . SMA causes progressive muscle weakness and atrophy, and significant unmet need remains, particularly in adults livi...
Source: Roche Investor Update - February 26, 2021 Category: Pharmaceuticals Source Type: news
Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older
Basel, 26 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi ™ (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease . SMA causes progressive muscle weakness and atrophy, and significant unmet need remains, particularly in adults livi...
Source: Roche Media News - February 26, 2021 Category: Pharmaceuticals Source Type: news
Roche announces results from Evrysdi (risdiplam) study in infants with Type 1 spinal muscular atrophy (SMA) published in New England Journal of Medicine
Basel, 25 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the New England Journal of Medicine has published Evrysdi ™ (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy (SMA). The data show that treatment with Evrysdi at 12 months helped 90% (19/21) of these infants survive without permanent ventilation and 33% (7/21) sit withou t support for at least 5 seconds, which is not normally seen in the natural course of the disease. The study also found that treatment with Evrysdi increased the levels of survival of motor...
Source: Roche Investor Update - February 25, 2021 Category: Pharmaceuticals Source Type: news
Roche announces results from Evrysdi (risdiplam) study in infants with Type 1 spinal muscular atrophy (SMA) published in New England Journal of Medicine
Basel, 25 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the New England Journal of Medicine has published Evrysdi ™ (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy (SMA). The data show that treatment with Evrysdi at 12 months helped 90% (19/21) of these infants survive without permanent ventilation and 33% (7/21) sit withou t support for at least 5 seconds, which is not normally seen in the natural course of the disease. The study also found that treatment with Evrysdi increased the levels of survival of motor...
Source: Roche Media News - February 25, 2021 Category: Pharmaceuticals Source Type: news
Invitation to Roche ’s virtual event on 24-month data of Evrysdi in patients with type 2 or type 3 spinal muscular atrophy presented at the 2021 MDA virtual conference
We are pleased to invite investors and analysts to participate in our virtual event on Friday, 19 March, 2021, highlighting Roche data presented during the 2021 Muscular Dystrophy Association - MDA -Virtual Clinical and Scientific Conference, from 15th to 18th February. (Source: Roche Investor Update)
Source: Roche Investor Update - February 19, 2021 Category: Pharmaceuticals Source Type: news
Bringing innovation to rare disease patients in Europe
Why is Biogen focusing on rare diseases? About 6,000 different rare diseases have been identified to date and these are estimated to affect 30 million people across Europe. It is essential that we collaborate across the medical community, industry, and governments to develop new therapies, explore new technologies and ensure equitable access to treatment. We must innovate across every aspect of treatment to transform the lives of people in these underserved communities. At Biogen, we have shown that pioneering in neuroscience can change the course of treatment for certain rare diseases. We have transformed the treatmen...
Source: EyeForPharma - February 17, 2021 Category: Pharmaceuticals Authors: Izzy Gladstone Source Type: news
#Icare4Rare campaign launches to raise awareness ahead of Rare Disease Week 2021
For many of the 30 million people affected by rare disease in the EU, lack of effective treatments and discrepancies in access and diagnosis are still common issues. Strong political will, innovation and further cooperation between countries remain vital to improve outcomes and transform their quality of life.To help raise awareness of rare disease, Reuters Events, Biogen and EU40 have collaborated to launch the #Icare4rare campaign to give prominence to Rare Disease Week 2021.Andrea Corazza, Head of Brussels Liaison Office, Public Policy& Government Affairs, Biogen, says: “With this campaign, we want to help putting...
Source: EyeForPharma - February 11, 2021 Category: Pharmaceuticals Authors: Izzy Gladstone Source Type: news
Roche reports solid results in 2020
Commenting on the Group ’s results, Roche CEO Severin Schwan said: “Roche continues to make important contributions to fighting the COVID-19 pandemic. We developed in record time a comprehensive portfolio of diagnostic solutions and entered new partnerships to develop and produce effective COVID-19 medicines. The deman d for our new medicines which benefit people living with serious conditions, such as cancer, multiple sclerosis, haemophilia and spinal muscular atrophy, remains high. Based on our rejuvenated portfolio and the significant progress made in developing our product pipeline, Roche is strongly positione d fo...
Source: Roche Media News - February 4, 2021 Category: Pharmaceuticals Source Type: news
Roche reports solid results in 2020
Commenting on the Group ’s results, Roche CEO Severin Schwan said: “Roche continues to make important contributions to fighting the COVID-19 pandemic. We developed in record time a comprehensive portfolio of diagnostic solutions and entered new partnerships to develop and produce effective COVID-19 medicines. The deman d for our new medicines which benefit people living with serious conditions, such as cancer, multiple sclerosis, haemophilia and spinal muscular atrophy, remains high. Based on our rejuvenated portfolio and the significant progress made in developing our product pipeline, Roche is strongly positione d fo...
Source: Roche Investor Update - February 4, 2021 Category: Pharmaceuticals Source Type: news
