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 Investor Update - 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 Media News - 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 Investor Update)
Source: Roche Investor Update - 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

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 Media News - 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 Investor Update - 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 Media News - 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 Investor Update - 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

Eight-week-old baby with spinal muscular atrophy could be saved by a single jab
Eight-week-old Edward, from Colchester, Essex, has the genetic condition spinal muscular atrophy (SMA), in which the lack of a protein called SMN results in progressively weaker muscles. (Source: the Mail online | Health)
Source: the Mail online | Health - December 15, 2020 Category: Consumer Health News Source Type: news

Overlooked No More: Barbara Waxman Fiduccia, Reproductive Rights Advocate
A sexual health educator and counselor in Los Angeles, she challenged a dominant culture that viewed people with disabilities as asexual beings. (Source: NYT Health)
Source: NYT Health - December 4, 2020 Category: Consumer Health News Authors: Denise Gellene Tags: Fiduccia, Barbara Waxman (1955-2001) Disabilities Discrimination Birth Control and Family Planning California Biographical Information Women and Girls Spinal Muscular Atrophy Source Type: news

Trinity researchers discover how the brain 're-wires' after disease
(Trinity College Dublin) Trinity College researchers are studying how the brain re-wires itself in neurological disease. The team is building treatments for today's more common global conditions like Motor Neurone Disease (MND/ALS) and Spinal Muscular Atrophy and their findings could impact rehabilitation for patients, the discovery of effective drugs and quantifying the potential efficacy of new therapies. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 24, 2020 Category: International Medicine & Public Health Source Type: news

Long-Term Benefits From Nusinersen for Presymptomatic SMA Long-Term Benefits From Nusinersen for Presymptomatic SMA
Nusinersen provides continued, long-term benefits to infants with spinal muscular atrophy who begin treatment before symptom onset, according to an analysis.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - October 23, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news

Chugai Files NDA for Risdiplam as the First Oral Drug for Spinal Muscular Atrophy in Japan
Dear Investor, Please find attached a press release by Chugai:https://www.roche.com/201015_IR_Chugai_eRisdiplam_NDA.pdf Do not hesitate to contact us for any further questions. With best regards, (Source: Roche Investor Update)
Source: Roche Investor Update - October 15, 2020 Category: Pharmaceuticals Source Type: news

Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi ™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve and achieve motor milestones. This exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. In addition, at two years, 59% (10/17 vs. 7/17 at 1-year) of infants were able to sit without support...
Source: Roche Media News - September 28, 2020 Category: Pharmaceuticals Source Type: news

Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi ™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve and achieve motor milestones. This exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. In addition, at two years, 59% (10/17 vs. 7/17 at 1-year) of infants were able to sit without support...
Source: Roche Investor Update - September 28, 2020 Category: Pharmaceuticals Source Type: news

SMA1 Infant Life-Saving Zolgensma Therapy Unavailable in Canada
Ground-breaking new drug Zolgensma that could stop progression of Spinal Muscular Atrophy Type 1 (SMA1) is the most expensive medication in the world (Source: Disabled World)
Source: Disabled World - September 15, 2020 Category: Disability Tags: Pharmaceutical Source Type: news

Can a rival to the world's most expensive drug help George with his muscle-wasting illness?
George Frost, 9, pictured with his sister Evie, left, was diagnosed with spinal muscular atrophy aged one. The youngster from Kent saw his muscles waste away until he was placed on a clinical trial. (Source: the Mail online | Health)
Source: the Mail online | Health - September 5, 2020 Category: Consumer Health News Source Type: news

Treatments for Inherited Neuromuscular Diseases of Childhood Treatments for Inherited Neuromuscular Diseases of Childhood
This review highlights recent advances in gene-specific therapies for neuromuscular diseases of childhood, including spinal muscular atrophy, Duchenne muscular dystrophy, and centronuclear myopathy.Seminars in Neurology (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - August 27, 2020 Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news

A multicenter look at gene therapy for spinal muscular atrophy
(Nationwide Children's Hospital) A new study confirms the safety and efficacy of gene therapy in children with spinal muscular atrophy under two years old. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - August 25, 2020 Category: Biology Source Type: news

FDA Approves First Oral Drug for Spinal Muscular Atrophy, Evrysdi (Risdiplam)
MONDAY, Aug. 10, 2020 -- The U.S. Food and Drug Administration has approved the first oral, at-home drug for spinal muscular atrophy (SMA), the agency announced Friday. Evrysdi (risdiplam) was approved to treat patients ages 2 months and older with... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - August 10, 2020 Category: General Medicine Source Type: news

FDA approves Roche ’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older
Roche announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi ™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. (Source: Roche Investor Update)
Source: Roche Investor Update - August 10, 2020 Category: Pharmaceuticals Source Type: news

FDA approves Roche ’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older
Roche announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi ™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. (Source: Roche Media News)
Source: Roche Media News - August 10, 2020 Category: Pharmaceuticals Source Type: news

FDA Approves Evrysdi (risdiplam) for Treatment of Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older
South San Francisco, CA -- August 7, 2020 -- Genentech, a member of the Roche Group, announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - August 7, 2020 Category: Drugs & Pharmacology Source Type: news

FDA OKs First Oral Treatment for Spinal Muscular Atrophy FDA OKs First Oral Treatment for Spinal Muscular Atrophy
The FDA has approved risdiplam (Evrysdi) as the first oral treatment for adults and children at least 2 months of age with spinal muscular atrophy.FDA Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - August 7, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news

Nusinersen with screening for spinal muscular atrophy cost effective?
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - June 30, 2020 Category: Drugs & Pharmacology 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 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

Treat early or wait? Experts ponder best way to manage milder forms of spinal muscular atrophy
(IOS Press) The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. Now, in a report published in the Journal of Neuromuscular Diseases, German researchers argue for an earlier start of treatment to prevent permanent nerve damage, challenging recommendations originally proposed by a group of American experts that suggests a strict follow-up strategy for children expected to have less severe disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 11, 2020 Category: International Medicine & Public Health Source Type: news

New longer-term data reinforce safety of Roche ’s satralizumab in adults and adolescents with neuromyelitis optica spectrum disorder
Basel, 22 May 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new pooled pivotal satralizumab safety results for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare, debilitating central nervous system disorder. These data are being presented at the 6th Annual Meeting of the European Academy of Neurology (EAN) and show satralizumab was well-tolerated in a broad patient population – including adolescents, for whom there is no approved medicine. Satralizumab is an investigational humanised monoclonal antibody that targets the interleukin-6 (IL-6) receptor, believed to play a key role in the i...
Source: Roche Investor Update - May 22, 2020 Category: Pharmaceuticals Source Type: news

New longer-term data reinforce safety of Roche ’s satralizumab in adults and adolescents with neuromyelitis optica spectrum disorder
Basel, 22 May 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new pooled pivotal satralizumab safety results for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare, debilitating central nervous system disorder. These data are being presented at the 6th Annual Meeting of the European Academy of Neurology (EAN) and show satralizumab was well-tolerated in a broad patient population – including adolescents, for whom there is no approved medicine. Satralizumab is an investigational humanised monoclonal antibody that targets the interleukin-6 (IL-6) receptor, believed to play a key role in the i...
Source: Roche Media News - May 22, 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
Source: Roche Media News - April 28, 2020 Category: Pharmaceuticals Source Type: news