Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders
Basel, 24 September 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced data from across its growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress 20 – 24 September 2021. The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to six weeks of age and data supporting the continued clinical investig ation of gene therapy, SRP-9001, in Duchenne muscular dystrophy (DMD).“These new data for Evrysdi may help extend the potential ben...
Source: Roche Investor Update - September 24, 2021 Category: Pharmaceuticals Source Type: news

Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders
Basel, 24 September 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced data from across its growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress 20 – 24 September 2021. The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to six weeks of age and data supporting the continued clinical investig ation of gene therapy, SRP-9001, in Duchenne muscular dystrophy (DMD).“These new data for Evrysdi may help extend the potential ben...
Source: Roche Media News - September 24, 2021 Category: Pharmaceuticals Source Type: news

Egypt: Egypt Launches Initiative for Early Detection of 19 Genetic Diseases in Newborns, Begins Free Treatment of Spinal Muscular Atrophy
[Egypt Online] Minister of Health and Population Hala Zayed announced on Tuesday 3/8/2021 the launch of an initiative for the early detection of genetic disorders and hereditary diseases in newly-born infants under the auspices of President Abdel Fattah El Sisi. (Source: AllAfrica News: Pregnancy and Childbirth)
Source: AllAfrica News: Pregnancy and Childbirth - August 4, 2021 Category: OBGYN 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.“With...
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.“With...
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 ...
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 ...
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

NICE does not recommend risdiplam for treating spinal muscular atrophy
NICE has published draft guidance for public consultation which does not recommend risdiplam (also called Evrysdi and made by Roche) for treating the rare genetic disorder spinal muscular atrophy (SMA). SMA is a progressive neuromuscular condition affecting the nerves in the spinal cord controlling movement. This leads to muscle weakness, progressive loss of movement, and difficulty breathing and swallowing. (Source: NHS Networks)
Source: NHS Networks - June 3, 2021 Category: UK Health Source Type: news

First NHS patient treated with gene therapy
A five-month old baby has become the first patient to receive a potentially life-saving drug on the NHS that can prevent paralysis and prolong the lives of children with Spinal Muscular Atrophy (SMA). Arthur Morgan, who was diagnosed with SMA earlier this month, received the one-off gene therapy at Evelina London Children ’s Hospital. (Source: NHS Networks)
Source: NHS Networks - June 2, 2021 Category: UK Health Source Type: news

Signs and Symptoms of Spinal Muscular Atrophy
What Are the Signs and Symptoms of Spinal Muscular Atrophy? (Source: eMedicineHealth.com)
Source: eMedicineHealth.com - April 20, 2021 Category: General Medicine Source Type: news

Is Spinal Muscular Atrophy Painful
? (Source: eMedicineHealth.com)
Source: eMedicineHealth.com - April 16, 2021 Category: General Medicine Source Type: news

New data for Roche ’s OCREVUS (ocrelizumab) reinforce significant benefit on slowing disease progression in relapsing and primary progressive multiple sclerosis
Basel, 16 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new OCREVUS ® (ocrelizumab) analyses supporting its significant benefit on disease progression in early-stage relapsing-remitting multiple sclerosis (RRMS) and primary progressive MS (PPMS) as well as demonstrating high persistence and strong adherence to twice-yearly (six-monthly) dosing. These data are being presented virtually at the 73rd American Academy of Neurology (AAN) Annual Meeting from 17–22 April 2021. OCREVUS is the number one prescribed MS medication in the U.S. for patients starting a new treatment, and more than 200,000 peop...
Source: Roche Media News - April 16, 2021 Category: Pharmaceuticals Source Type: news

New data for Roche ’s OCREVUS (ocrelizumab) reinforce significant benefit on slowing disease progression in relapsing and primary progressive multiple sclerosis
Basel, 16 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new OCREVUS ® (ocrelizumab) analyses supporting its significant benefit on disease progression in early-stage relapsing-remitting multiple sclerosis (RRMS) and primary progressive MS (PPMS) as well as demonstrating high persistence and strong adherence to twice-yearly (six-monthly) dosing. These data are being presented virtually at the 73rd American Academy of Neurology (AAN) Annual Meeting from 17–22 April 2021. OCREVUS is the number one prescribed MS medication in the U.S. for patients starting a new treatment, and more than 200,000 peop...
Source: Roche Investor Update - April 16, 2021 Category: Pharmaceuticals Source Type: news

Roche ’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA)
Basel, 15 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi ™ (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to sit without support. The study also showed Evrysdi continued to improve survi val, improve ability to feed orally and reduce the need for permanent ventilation*. Exploratory data suggested Evrysdi continued to improve the ability to swallow and...
Source: Roche Investor Update - April 15, 2021 Category: Pharmaceuticals Source Type: news

Roche ’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA)
Basel, 15 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi ™ (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to sit without support. The study also showed Evrysdi continued to improve survi val, improve ability to feed orally and reduce the need for permanent ventilation*. Exploratory data suggested Evrysdi continued to improve the ability to swallow and...
Source: Roche Media News - April 15, 2021 Category: Pharmaceuticals Source Type: news

New Roche data at 2021 AAN highlight impact and breadth of expanding neuroscience portfolio
             Basel, 8 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data for its approved and investigational medicines for the treatment of neurological disorders will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021. These new data include 23 abstracts highlighting the expanding Roche neuroscience portfolio across six therapeutic areas, including EVRYSDI ™ (risdiplam) for spinal muscular atrophy (SMA), OCREVUS® (ocrelizumab) in relapsing and primary progre...
Source: Roche Media News - April 8, 2021 Category: Pharmaceuticals Source Type: news

New Roche data at 2021 AAN highlight impact and breadth of expanding neuroscience portfolio
             Basel, 8 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data for its approved and investigational medicines for the treatment of neurological disorders will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021. These new data include 23 abstracts highlighting the expanding Roche neuroscience portfolio across six therapeutic areas, including EVRYSDI ™ (risdiplam) for spinal muscular atrophy (SMA), OCREVUS® (ocrelizumab) in relapsing and primary progre...
Source: Roche Investor Update - April 8, 2021 Category: Pharmaceuticals Source Type: news

How Do You Diagnose Spinal Muscular Atrophy?
Title: How Do You Diagnose Spinal Muscular Atrophy?Category: Diseases and ConditionsCreated: 4/7/2021 12:00:00 AMLast Editorial Review: 4/7/2021 12:00:00 AM (Source: MedicineNet Kids Health General)
Source: MedicineNet Kids Health General - April 7, 2021 Category: Pediatrics Source Type: news

Spinal Muscular Atrophy Drug Approved in Europe Spinal Muscular Atrophy Drug Approved in Europe
The European Commission (EC) has granted marketing clearance for risdiplam, the first oral drug for patients with certain types of spinal muscular atrophy.Medscape Medical News (Source: Medscape Internal Medicine Headlines)
Source: Medscape Internal Medicine Headlines - March 30, 2021 Category: Internal Medicine Tags: Neurology & Neurosurgery News Source Type: news

PTC Therapeutics Announces the European Approval of Evrysdi(TM) for the Treatment of Spinal Muscular Atrophy
First and only at home treatment approved for SMA Proven efficacy in adults and children with SMA 2 months and older SOUTH PLAINFIELD, N.J., March 30, 2021 -- (Healthcare Sales & Marketing Network) -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today anno... Biopharmaceuticals, Regulatory PTC Therapeutics, Evrysdi, risdiplam, spinal muscular atrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - March 30, 2021 Category: Pharmaceuticals Source Type: news

Roche ’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy
Basel, 30 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has approved Evrysdi ™ (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients two 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 disea se. The condition causes muscle weakness and progressive loss of movement and significant unmet need remains, particularly in adults living with this condition. (Source: Roche Media News)
Source: Roche Media News - March 30, 2021 Category: Pharmaceuticals Source Type: news

Roche ’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy
Basel, 30 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has approved Evrysdi ™ (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients two 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 disea se. The condition causes muscle weakness and progressive loss of movement and significant unmet need remains, particularly in adults living with this condition. (Source: Roche Investor Update)
Source: Roche Investor Update - March 30, 2021 Category: Pharmaceuticals Source Type: news

Drug for spinal muscular atrophy disease already exempt from import duty: FM
Giving a clarification on the issue raised by Congress Vivek K Tankha during Zero Hour on March 17, Sitharaman in Rajya Sabha said the member had day before yesterday stated that there is one medicine to treat spinal muscular atrophy disease which costs about Rs 16 crore and there is a tax component at Rs 7 crore. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - March 19, 2021 Category: Pharmaceuticals Source Type: news

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. Th...
Source: Roche Investor Update - March 16, 2021 Category: Pharmaceuticals Source Type: news

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. Th...
Source: Roche Media News - 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 ...
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 ...
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 m...
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 m...
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 ...
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 put...
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 positi...
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 positi...
Source: Roche Investor Update - February 4, 2021 Category: Pharmaceuticals Source Type: news