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
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NICE has published draft guidance for public consultation which does not recommend risdiplam (also called Evrysdi and made by Roche) for treating the rare genetic disorder spinal muscular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 people have...
Source: Roche Investor Update - 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 people have...
Source: Roche Media News - 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 red...
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 red...
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 progressive multiple sclerosis (RMS and PPMS), investigational Br...
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 progressive multiple sclerosis (RMS and PPMS), investigational Br...
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
