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 for...
Source: Roche Investor Update - 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 for...
Source: Roche Media News - 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
