Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)
Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam le...
Source: Roche Investor Update - June 12, 2020 Category: Pharmaceuticals Source Type: news
Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)
Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam le...
Source: Roche Media News - June 12, 2020 Category: Pharmaceuticals Source Type: news
Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p<0.0001) sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). No infants achieve this milestone in the natural history of Type 1 SMA. In addition, 18 (43.9%) infants were able to hold their head upright...
Source: Roche Investor Update - April 28, 2020 Category: Pharmaceuticals Source Type: news
Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p<0.0001) sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). No infants achieve this milestone in the natural history of Type 1 SMA. In addition, 18 (43.9%) infants were able to hold their head upright...
Source: Roche Media News - April 28, 2020 Category: Pharmaceuticals Source Type: news
'I feel like I don't count,' says man with MND
The government's advice on shielding is failing people with motor neurone disease, according to a charity. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - April 26, 2020 Category: Consumer Health News Source Type: news
Coronavirus: Lockdown, a baby and MND - but I'm feeling great
Lucy Lintott is the youngest person in Scotland with motor neurone disease - and is now facing a mountain of new challenges. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - April 9, 2020 Category: Consumer Health News Source Type: news
Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)
Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by August 24, 2020. The extension is a result of the recent submission of additional data by Roche, including data from the pivotal SUNFISH Part 2 study, in close collaboration with the FDA. These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy. In November 2019, the FDA granted Priority Rev...
Source: Roche Media News - April 7, 2020 Category: Pharmaceuticals Source Type: news
Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)
Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by August 24, 2020. The extension is a result of the recent submission of additional data by Roche, including data from the pivotal SUNFISH Part 2 study, in close collaboration with the FDA. These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy. In November 2019, the FDA granted Priority Rev...
Source: Roche Investor Update - April 7, 2020 Category: Pharmaceuticals Source Type: news
GP surgery sends out letter asking terminally ill patients to sign DNR notices
Llynfi Surgery, in Maesteg, South Wales, sent the letter to patients with serious health conditions such as incurable cancer, motor neurone disease, and untreatable heart and lung conditions. (Source: the Mail online | Health)
Source: the Mail online | Health - April 1, 2020 Category: Consumer Health News Source Type: news
New brain reading technology could help the development of brainwave-controlled devices
(The Francis Crick Institute) A new method to accurately record brain activity at scale has been developed by researchers at the Crick, Stanford University and UCL. The technique could lead to new medical devices to help amputees, people with paralysis or people with neurological conditions such as motor neurone disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 20, 2020 Category: International Medicine & Public Health Source Type: news
Coronavirus UK: Death toll hits 137, youngest fatality is 47
Only a handful of coronavirus victims have already been identified, including the UK's youngest - a 45-year-old with motor neurone disease from Kettering in Northamptonshire. (Source: the Mail online | Health)
Source: the Mail online | Health - March 19, 2020 Category: Consumer Health News Source Type: news
Coronavirus: Craig Ruston 'youngest UK death'
Craig Ruston, who had motor neurone disease, died on Monday morning in Kettering. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - March 18, 2020 Category: Consumer Health News Source Type: news
Experts discover toolkit to repair DNA breaks linked to aging, cancer and MND
(University of Sheffield) A new 'toolkit' to repair damaged DNA that can lead to aging, cancer and motor neurone disease (MND) has been discovered by scientists at the universities of Sheffield and Oxford. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - March 9, 2020 Category: Biology Source Type: news
Initial protective role of nervous system's 'star-shaped cells' in sporadic motor neuron disease uncovered
(The Francis Crick Institute) Support cells in the nervous system help protect motor neurons in the early-stages of sporadic motor neuron disease, according to new research from the Crick and UCL. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 10, 2020 Category: International Medicine & Public Health Source Type: news
Roche ’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy
Basel, 23 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was consistent with its known safety profile and no new saf...
Source: Roche Media News - January 23, 2020 Category: Pharmaceuticals Source Type: news
