Data for Roche ’s Evrysdi® (risdiplam) published in New England Journal of Medicine shows significant improvement in survival and motor milestones in babies with Type 1 spinal muscular atrophy (SMA)
Basel, 29 July 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the New England Journal of Medicine (NEJM) has published data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies aged 1-7 months old with symptomatic Type 1 spinal muscular atr ophy (SMA). The study met its primary endpoint with 29% of infants (12/41) sitting without support for at least five seconds* by month 12, a milestone not seen in the natural course of the disease. Safety for Evrysdi in the FIREFISH Part 2 study was consistent with its known safety profile.“Without treatme...
Source: Roche Media News - July 29, 2021 Category: Pharmaceuticals Source Type: news
Data for Roche ’s Evrysdi® (risdiplam) published in New England Journal of Medicine shows significant improvement in survival and motor milestones in babies with Type 1 spinal muscular atrophy (SMA)
Basel, 29 July 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the New England Journal of Medicine (NEJM) has published data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies aged 1-7 months old with symptomatic Type 1 spinal muscular atr ophy (SMA). The study met its primary endpoint with 29% of infants (12/41) sitting without support for at least five seconds* by month 12, a milestone not seen in the natural course of the disease. Safety for Evrysdi in the FIREFISH Part 2 study was consistent with its known safety profile.“Without treatme...
Source: Roche Investor Update - July 29, 2021 Category: Pharmaceuticals Source Type: news
Roche launches spinal muscular atrophy treatment drug in India priced at around Rs 6 lakh per bottle
Since its launch, over 4,000 SMA patients across over 50 countries have benefitted from the drug, Roche said in a statement. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - July 29, 2021 Category: Pharmaceuticals Source Type: news
Scrap taxes on drug import for spinal muscular atrophy, Stalin tells Centre
For children affected by SPA, gene-therapy has to be ideally given before the child reaches two years of age and this therapy costs over Rs 16 crore per person, Stalin said in a letter to union finance minister Nirmala Sitharaman. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - July 13, 2021 Category: Pharmaceuticals Source Type: news
Baby gets go-ahead for world ’s most expensive drug from NHS
NHS approved to use gene therapy to treat baby born with spinal muscular atrophyThe parents of a baby with a fatal condition have succeeded in their campaign for their son to be treated with the world ’s most expensive drug.A new gene therapy, Zolgensma, will be used to treat 10-month-old Edward, from Colchester, who has severe spinal muscular atrophy (SMA), after his parents were given the green light earlier this week. The genetic condition, which is caused by a missing protein, weakens the muscles and affects movement and breathing.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - July 11, 2021 Category: Science Authors: Clea Skopeliti Tags: NHS Medical research Health Society Science UK news Source Type: news
Sharing Mayo Clinic: Every day you don't lose, you are winning
This article is written by Mayo Clinic Staff. Jennifer Summers, is an optimistic, articulate woman. The 35-year-old enjoys crafting, spending time with her close-knit… (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - July 11, 2021 Category: Databases & Libraries Source Type: news
World Rallies for Little Girl to Purchase $2.1 Million Drug
A young girl with spinal muscular atrophy receives tremendous support in the race to purchase the drug Zolgensma before she turns 2. (Source: WebMD Health)
Source: WebMD Health - July 9, 2021 Category: Consumer Health News Source Type: news
Gait-assisted exoskeletons for children with cerebral palsy or spinal muscular atrophy: a systematic review - Cumplido C, Delgado E, Ramos J, Puyuelo G, Garc és E, Destarac MA, Plaza A, Hernández M, Gutiérrez A, Garcia E.
BACKGROUND: Cerebral Palsy (CP) and Spinal Muscular Atrophy (SMA) are common causes of motor disability in childhood. Gait exoskeletons are currently being used as part of rehabilitation for children with walking difficulties. OBJECTIVE: To assess ... (Source: SafetyLit)
Source: SafetyLit - July 8, 2021 Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news
New components for antisense gene therapy show promise in treating spinal muscular atrophy
(Skolkovo Institute of Science and Technology (Skoltech)) Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease.The new findings will help develop drugs with less toxicity and fewer injections needed thanks to prolonged action. Nusinersen, an approved drug for the treatment of SMA, is administered several times a year via an injection into the spinal canal, so fewer injections would improve the quality of life for patients with SMA. (Source: EurekAlert! -...
Source: EurekAlert! - Medicine and Health - June 29, 2021 Category: International Medicine & Public Health Source Type: news
Chugai obtains regulatory approval for Evrysdi for the treatment of Spinal Muscular Atrophy
Dear Investor,Please find attached a press release by Chugai:https://www.roche.com/210623_IR_Chugai_eEvrysdi_approval.pdfDo not hesitate to contact us for any further questions.With best regards, (Source: Roche Investor Update)
Source: Roche Investor Update - June 23, 2021 Category: Pharmaceuticals Source Type: news
Evrysdi(TM) Approved in Japan for the Treatment of Spinal Muscular Atrophy
48th country that approved Evrysdi
First commercial sale in Japan will trigger $10M milestone payment from Roche
SOUTH PLAINFIELD, N.J., June 23, 2021 -- (Healthcare Sales & Marketing Network) -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced... Biopharmaceuticals, Regulatory PTC Therapeutics, Evrysdi, risdiplam, spinal muscular atrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 23, 2021 Category: Pharmaceuticals Source Type: news
Roche data at EAN 2021 showcase significant impact of therapies across diverse neuroscience portfolio
Basel, 15 June 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that data across its growing neuroscience portfolio will be presented at the 7th Congress of the European Academy of Neurology (EAN) Annual Meeting being held virtually 19-22 June, 2021. These new and encore data demonstrate Roche ’s commitment to advancing the clinical understanding of a broad range of neurological disorders with the goal of meeting the needs of people living with both the rarest and most common conditions.“Our data at EAN and recent European regulatory milestones for EVRYSDI and ENSPRYNG reflect our continued commitment to disco...
Source: Roche Media News - June 15, 2021 Category: Pharmaceuticals Source Type: news
Roche data at EAN 2021 showcase significant impact of therapies across diverse neuroscience portfolio
Basel, 15 June 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that data across its growing neuroscience portfolio will be presented at the 7th Congress of the European Academy of Neurology (EAN) Annual Meeting being held virtually 19-22 June, 2021. These new and encore data demonstrate Roche ’s commitment to advancing the clinical understanding of a broad range of neurological disorders with the goal of meeting the needs of people living with both the rarest and most common conditions.“Our data at EAN and recent European regulatory milestones for EVRYSDI and ENSPRYNG reflect our continued commitment to disco...
Source: Roche Investor Update - June 15, 2021 Category: Pharmaceuticals Source Type: news
New Roche data for Evrysdi show improved motor function in pre-symptomatic babies after one year and confirm safety profile in previously treated people with spinal muscular atrophy (SMA)
Basel, 11 June 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new interim data from two studies of Evrysdi ® (risdiplam); JEWELFISH and RAINBOWFISH. Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated with another SMA-targeting therapy, including nusinersen and onasemnogene abeparvovec, showed the safet y profile of Evrysdi and increase in SMN protein levels are consistent with those observed in other Evrysdi studies. (Source: Roche Investor Update)
Source: Roche Investor Update - June 11, 2021 Category: Pharmaceuticals Source Type: news
New Roche data for Evrysdi show improved motor function in pre-symptomatic babies after one year and confirm safety profile in previously treated people with spinal muscular atrophy (SMA)
Basel, 11 June 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new interim data from two studies of Evrysdi ® (risdiplam); JEWELFISH and RAINBOWFISH. Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated with another SMA-targeting therapy, including nusinersen and onasemnogene abeparvovec, showed the safet y profile of Evrysdi and increase in SMN protein levels are consistent with those observed in other Evrysdi studies. (Source: Roche Media News)
Source: Roche Media News - June 11, 2021 Category: Pharmaceuticals Source Type: news
