New three-year data for Roche ’s Evrysdi (risdiplam) show long-term improvements in survival and motor milestones in babies with Type 1 spinal muscular atrophy (SMA)
91% of infants treated withEvrysdi in the FIREFISH study were still alive at three yearsInfants treated withEvrysdi maintained or continued to improve in measures of motor function, including their ability to sit without support for 5 and 30 secondsEvrysdi has proven efficacy in infants and adults, with over 5,000 patients treated to dateBasel, 29 April 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new three-year data from the FIREFISH study, including one-year data from the open label extension, reinforcing the long-term efficacy and safety of Evrysdi ® (risdiplam) in infants with symptomatic Type 1 spinal mu...
Source: Roche Investor Update - April 29, 2022 Category: Pharmaceuticals Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche reports good sales growth in the first quarter of 2022
Basel, 25 April 2022Group sales increase 11%1 at constant exchange rates (CER) and 10% in Swiss francsPharmaceuticals Divisionsales up 6%; continued strong sales of new medicines for severe diseases. Impact of biosimilars decreases as expected.Diagnostics Divisionsales grow 24% due to good momentum in base business and continued high demand for COVID-19 tests. After a strong first quarter, significant decline in COVID-19-related testing expected.Highlights in the first quarter:FDA approval of Vabysmo (severe eye diseases); CHMP recommends EU approval of Polivy combination (aggressive form of blood cancer), Tecentriq (early...
Source: Roche Media News - April 25, 2022 Category: Pharmaceuticals Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche reports good sales growth in the first quarter of 2022
Group sales increase 11%1 at constant exchange rates (CER) and 10% in Swiss francsPharmaceuticals Divisionsales up 6%; continued strong sales of new medicines for severe diseases. Impact of biosimilars decreases as expected.Diagnostics Divisionsales grow 24% due to good momentum in base business and continued high demand for COVID-19 tests. After a strong first quarter, significant decline in COVID-19-related testing expected.Highlights in the first quarter:FDA approval of Vabysmo (severe eye diseases); CHMP recommends EU approval of Polivy combination (aggressive form of blood cancer), Tecentriq (early-stage non-small cel...
Source: Roche Investor Update - April 25, 2022 Category: Pharmaceuticals Source Type: news
In India, Parents of Children with Rare Disease Plea for Help Online
India makes many of the world ’s drugs, but treatments for rare diseases like spinal muscular atrophy are imported and prohibitively costly. In desperation, parents are raising funds on social media. (Source: NYT Health)
Source: NYT Health - April 6, 2022 Category: Consumer Health News Authors: Sameer Yasir, Shalini Venugopal Bhagat and Sara Hylton Tags: India Spinal Muscular Atrophy Drugs (Pharmaceuticals) Crowdfunding (Internet) Parenting Biogen Inc Novartis AG Roche Holding AG Babies and Infants Children and Childhood Source Type: news
Cell & Gene Therapy: High stakes and high hopes for healthcare
Revolutionary cell and gene therapies (CGT) delivering breakthrough treatments have begun to disrupt healthcare. The companies building these next-generation pharma technologies are introducing radically new operating models, influencing industry development and attracting large-scale media and investor attention. However, while growing rapidly, CGT is expected to see around just $34 billion of sales in 2030, up from $4.4 billion in 2020 compared to the global industry figure of £1.27 trillion in 2020. Although there are hundreds of CGT treatments in the pipeline, the challenges have so far kept the approved treatments ...
Source: EyeForPharma - March 29, 2022 Category: Pharmaceuticals Authors: Pamela Whitby Source Type: news
Roche to present data across broad and impactful neuroscience portfolio at 2022 AAN Annual Meeting
First data showing OCREVUS treatment effect on disability progression in non-active secondary progressive multiple sclerosis and further data in primary progressive MS will be presentedEvrysdi data continue to demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophyLonger-term efficacy and safety forEnspryng inneuromyelitisoptica spectrum disorder reinforce previously seen resultsAdditional data across neurological disorders, including Alzheimer ’s disease, help advance the scientific understanding of these conditions and the potential impact of early treatmentBasel, 25 March...
Source: Roche Investor Update - March 25, 2022 Category: Pharmaceuticals Source Type: news
Roche to present data across broad and impactful neuroscience portfolio at 2022 AAN Annual Meeting
First data showing OCREVUS treatment effect on disability progression in non-active secondary progressive multiple sclerosis and further data in primary progressive MS will be presentedEvrysdi data continue to demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophyLonger-term efficacy and safety forEnspryng inneuromyelitisoptica spectrum disorder reinforce previously seen resultsAdditional data across neurological disorders, including Alzheimer ’s disease, help advance the scientific understanding of these conditions and the potential impact of early treatmentBasel, 25 March...
Source: Roche Media News - March 25, 2022 Category: Pharmaceuticals Source Type: news
Thames Valley pilot study to check for rare condition
Babies born with spinal muscular atrophy (SMA) have better prospects if it is picked up at birth. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - March 17, 2022 Category: Consumer Health News Source Type: news
New data for Roche ’s Evrysdi (risdiplam) demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophy (SMA)
Long-term efficacy data from the pivotal SUNFISH study confirm increases in motor function are sustained at three years while adverse events decreased over the same periodPart 2 of SUNFISH showedEvrysdi demonstrated a marked improvement in, orstabilisation of, motor function after two years compared to an untreated external control groupLatest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated withEvrysdi for at least 12 months were able to stand and walk within timeframes typical of healthy babiesMore than 5,000 patients have been treated withEvrysdi to date, from newborns to people...
Source: Roche Investor Update - March 16, 2022 Category: Pharmaceuticals Source Type: news
New data for Roche ’s Evrysdi (risdiplam) demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophy (SMA)
Long-term efficacy data from the pivotal SUNFISH study confirm increases in motor function are sustained at three years while adverse events decreased over the same periodPart 2 of SUNFISH showedEvrysdi demonstrated a marked improvement in, orstabilisation of, motor function after two years compared to an untreated external control groupLatest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated withEvrysdi for at least 12 months were able to stand and walk within timeframes typical of healthy babiesMore than 5,000 patients have been treated withEvrysdi to date, from newborns to people...
Source: Roche Media News - March 16, 2022 Category: Pharmaceuticals Source Type: news
Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio
Evrysdi ® (risdiplam) data further confirm long-term efficacy and safety data in a broad range of people with spinal muscular atrophy (SMA)Latest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated with Evrysdi for at least 12 months were able to sit, stand and walk within timeframes typical of healthy babiesNew gene therapy data in boys living with Duchenne muscular dystrophy (DMD) reports encouraging results from early stage study supporting its progress into global pivotal Phase III EMBARK studyBasel, 08 March 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data...
Source: Roche Media News - March 8, 2022 Category: Pharmaceuticals Source Type: news
Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio
Evrysdi ® (risdiplam) data further confirm long-term efficacy and safety data in a broad range of people with spinal muscular atrophy (SMA)Latest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated with Evrysdi for at least 12 months were able to sit, stand and walk within timeframes typical of healthy babiesNew gene therapy data in boys living with Duchenne muscular dystrophy (DMD) reports encouraging results from early stage study supporting its progress into global pivotal Phase III EMBARK studyBasel, 08 March 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data...
Source: Roche Investor Update - March 8, 2022 Category: Pharmaceuticals Source Type: news
‘It’s a real-life Hunger Games’: a lifesaving drug costs $2m, but not every child can get it
Zolgensma is a revolutionary gene therapy that can stop a deadly childhood condition called SMA in its tracks. It ’s also one of the most expensive drugs in the worldElizabeth Wraige remembers the first time she delivered the diagnosis. “Parents feel as if they’ve been hit by a sledgehammer,” she says. Her patient, a baby boy, had been born tiny and perfect to overjoyed parents six months earlier. But they had begun to feel something was not right. He seemed floppy and was not moving normally.Tests showed he had spinal muscular atrophy (SMA), themost deadly genetic condition in children under two, in whicha deficit...
Source: Guardian Unlimited Science - March 5, 2022 Category: Science Authors: Hannah Devlin. Photographs: Paolo Woods. Captions: Arnaud Robert Tags: Pharmaceuticals industry Neuroscience Medical research Drugs Genetics Health NHS Health policy Parents and parenting Family Life and style Source Type: news
Roche and three leading research institutions combine expertise on new Alzheimer ’s prevention trial
Phase III trial will evaluategantenerumab in cognitively unimpaired people with the earliest biologicalsigns ofAlzheimer ' sdiseaseRoche and research institutions have jointly leveraged their scientific expertise on Alzheimer ’s disease to design the new studyBasel, 3 March 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced a new Phase III Alzheimer ’s disease prevention trial (SKYLINE, NCT05256134) with gantenerumab, an investigational anti-amyloid antibody that is administered subcutaneously. Roche intends to enter into a collaboration agreement with Banner Alzheimer’s Institute’s Alzheimer’s Prevention ...
Source: Roche Investor Update - March 3, 2022 Category: Pharmaceuticals Source Type: news
Biohaven Licenses Taldefgrobep Alfa, a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA), from Bristol Myers Squibb
Biohaven acquires worldwide rights to phase 3-ready neuromuscular program
Taldefgrobep alfa expands Biohaven's portfolio of innovative, late-stage product candidates for the treatment of neurologic, neuroinflammatory, and neuropsychiatric disorders
N... Biopharmaceuticals, Licensing Biohaven Pharmaceutical, Bristol Myers Squibb, taldefgrobep alfa (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - February 25, 2022 Category: Pharmaceuticals Source Type: news
