Spinal muscular atrophy

Six-month-old boy has just months to live after the NHS refuse to pay for a ‘miracle drug’ – Daily Mail

A six-month-old boy with a devastating genetic condition has been given just three months to live unless…

0294

This gorgeous baby boy has only three months to live unless the NHS decides to fund a miracle drug – Manchester Evening News

Lying in the Intensive Care Unit of Royal Manchester Children’s Hospital, baby Haris Khan listens to some…

0525

British MP Pushes to Make Spinraza Treatment Available for SMA Patients – SMA News Today

Citing the deteriorating health of a child with spinal muscular atrophy (SMA), a member of the British…

0452

Scotland Opens Spinraza to All SMA Patients Using Its Public Health System – SMA News Today

Scotland has joined the growing roster of countries making the spinal muscular atrophy (SMA) therapy Spinraza available…

0391

Drugs company urged to ‘do the decent thing’ and help Forest Gate baby facing a death sentence – Newham Recorder

Lyn Brown spoke out in parliament this week about the heartache a family from Forest Gate face…

0463

Our Family Photo Album Sparks Memories of SMA Challenges – SMA News Today

It was time to take down the Christmas decorations. Every room was filled with boxes and coffee…

0463

Couple tackle disability ‘misconceptions’ with YouTube channel – Belfast Telegraph

A man with muscular dystrophy and his able-bodied girlfriend are sharing their relationship with the world on…

0672

Pitching In When a Family Member Is Out of Commission – SMA News Today

It takes a family to navigate SMA. All five of us play a role in helping the…

0548

Technology Has Improved Quality of Life for Those with SMA – SMA News Today

Technology is a wonderful thing. It’s something that is a part of all of our lives and…

0699

Disabled man and able-bodied girlfriend want to show their love to the world – Mirror Online

A disabled man and his able-bodied girlfriend want to show off their love to the world. Shane Burcaw,…

0771

Top 10 SMA Stories of 2018 – SMA News Today

SMA News Today brought you daily coverage of important findings, key treatment developments and clinical trials related…

0641

Roche drug shows benefit in spinal muscular atrophy – BioPharma Dive

Dive Brief: Fresh results from two studies of Roche’s experimental oral therapy for spinal muscular atrophy (SMA) support…

0699

Babies with spinal muscular atrophy to be covered for life-saving but costly Spinraza drug – The Globe and Mail

Canadian children with a rare and often fatal neuromuscular disorder are about to gain public funding for…

0723

Northampton mum’s plea to NHS to fund rare drug to change son’s life – Northampton Chronicle and Echo

A Northampton mum-of-one is urging the NHS to pay for a rare drug needed to keep her…

01.3k

Mother in NHS plea for spinal muscular atrophy drug – BBC News

A mother whose six-year-old son has a rare muscle wasting disease says his future is dark if…

01.4k

Derby twins, 13, make desperate plea for new drug that could prolong their lives – Derbyshire Live

A disabled boy says he and his twin brother should be given access to a new drug…

01.1k

Why are our children being denied life saving treatment? – Manchester Evening News

Catching the slightest cold could put Jacob O’Neill in hospital for three weeks. He suffers from Spinal Muscular…

01.5k

Outrage as ‘miracle’ drug too expensive for NHS – The Argus

A “MIRACLE” drug which could help thousands of people with a lifelong genetic condition is not going…

01.5k

Surf dog Ricochet paddles out with kids suffering from spinal muscular atrophy – CBS 8 San Diego

Ricochet the surf dog is hanging paw again, this time he is surfing with kids suffering from…

01.8k

Spinal muscular atrophy: How can recent breakthroughs get patients moving again? – Medical News Bulletin

A review of the current treatment options for spinal muscular atrophy is presented by a UK team…

01.6k

The case of Spinal Muscular Atrophy: Deadly inherited diseases start to yield – Tri Corner News

Part I of 2 A few years ago, a friend asked me if I could take over a…

01.6k

Teenager with degenerative muscle disease struggles to swallow food or even cough – Bristol Live

A teenager who suffers from a rare degenerative muscle disease struggles to cough or even swallow his…

01.8k

Instagram blogger inspires others with disabilities to enjoy colourful fashion – The Independent

The fashion industry has been making strides in recent years to become more inclusive and diverse. However, it…

02k

Scientists investigate new strategy to treat spinal muscular atrophy in infants – The Scripps Research Institute

Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble…

02k

Scientists investigate new strategy to treat spinal muscular atrophy in infants – Medical Xpress

Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble…

02.1k

Health chiefs urged to approve life-changing drug for Muscular Dystrophy patients – The Scottish Sun

PATIENTS with a genetic condition could lose the ability to move, breathe and swallow unless health bosses…

02.4k

Bath Half Marathon: These inspirational people from Midsomer Norton and Radstock are running in aid of local charities – Somerset Live

Local people are currently gearing up to prepare for the Bath Half Marathon – and it is…

02.4k

Mother’s plea for drug which could help her disabled son – ITV News

A mother from Welwyn Garden City is urging the NHS to allow the use of a drug…

02.6k

Spinal muscular atrophy (SMA) patients could get access to drug earlier than expected – Daily Mail

Patients with the devastating genetic condition, spinal muscular atrophy (SMA), could gain access to vital treatment earlier…

02.7k

Have Scientists Found a Treatment for Spinal Muscular Atrophy? – Medical News Bulletin

Spinal muscular atrophy often leads to an inability to breathe or death. Researchers tested a new gene therapy for the disease. Spinal muscular atrophy…

02.4k

Most people in favor of screening for spinal muscular atrophy – Science Daily

Research from the University of Warwick indicates that most people are in favour of newborn screening for the potentially deadly condition spinal muscular…

02.4k

‘I felt like second-class citizen when British Airways denied me boarding’, disabled composer says – Evening Standard

Evening Standard

02.4k

Nusinersen Improves Motor Function, Survival in Infants With Spinal Muscular Atrophy – Neurology Advisor

Infants with spinal muscular atrophy who receive nusinersen experience greater survival and motor function improvement compared with controls, according to findings from a…

02.3k

Gene therapy could cure Spinal Muscular Atrophy – ABC Online

A criticism is often levelled at pharmaceutical companies that they don’t seek to cure diseases – they only seek to find a long-term…

02.3k

New drug enables infants with genetic disorder to live longer, gain motor function – Science Daily

Infants with the most severe form of spinal muscular atrophy (SMA) were more likely to show gains in motor function and were 47…

02.2k

Most would-be parents who carry severe genetic disorders are unaware – The Age

Nearly 90 per cent of couples at risk of having babies with devastating genetic conditions have no family history of the disorders and…

02.4k

Acadiana family fighting to find a cure for Spinal Muscular Atrophy – KATC Lafayette News

Spinal Muscular Atrophy is a genetic disorder that affects the control of muscle movement. It affects crawling, walking,…

02.3k

Family fundraising for new wheelchair so 3-year-old girl can go to school – Bath Chronicle

Family and friends are fundraising to buy a three-year-old girl a new wheelchair for her birthday. Isabel Freeman from Midsomer Norton is unable to…

02.1k

Charity’s fears over cuts applied to patient’s healthcare budget – Norfolk Eastern Daily Press

Muscular Dystrophy UK has told Norwich Clinical Commissioning Group (CCG) its patients deserve clarity after 39-year-old Kirsty Read looked into the money she…

02.1k

Stratford District Council retains Disability Confident Employer status – Stratford upon Avon Herald

STRATFORD-ON-AVON District Council has retained its Disability Confident Employer status until 2019. To achieve this standard the council has shown a commitment to support…

01.9k

‘We were told to say goodbye to Noah and then had to fight to get a life-saving drug… now we want to give something back’ – Belfast Telegraph

Like most parents Jonny Collins (36) from Lisburn thinks he has the happiest, best four-year-old child in the world. But Jonny, who is…

02.5k

Iowa State University biomedical researcher conducts promising trial of potential therapy for spinal muscular atrophy – Iowa State University

A drug developed by an Iowa State University biomedical researcher as a potential treatment for spinal muscular atrophy showed promising results in a…

02.1k

11 Fast Facts About Spinal Muscular Atrophy – SMA News Today

How much do you know about spinal muscular atrophy (SMA)? It’s a genetic disease that progressively weakens the muscles of babies and small…

02.1k

Spinal muscular atrophy drug gives new hope for babies with cruel disease – ABC Online

Spinal muscular atrophy, or SMA, is one of those diseases you probably will not hear about until it affects someone you love. Then…

02.1k

Desktop 3D Printer Produces Arm Brace for Child with SMA – Machine Design

A child living in Poland was the recipient of a 3D-printed hand exoskeleton that would help him to perform everyday tasks while living…

01.9k

‘We don’t know how long we’ve got her for’: First-time parents share anguish as they spend their final moments with their terminally ill baby daughter born with a rare muscle-wasting disease – Daily Mail

A young couple has opened up about their anguish as they spend whatever time they have left with their terminally ill baby daughter. Sydney…

02k

Family’s debt because they can’t pay electric bill to keep daughter alive – Metro

A family say they are running into debt because of the high energy bills to keep their daughter alive. Maddison Sherwood has a rare…

01.9k

Family of girl, 8, struggling to pay electricity bill for equipment to keep her alive – Nottingham Post

The family of an eight-year-old girl say they are running into debt with their power provider after their monthly bill rocketed due to…

02k

Polish Designer Uses Sinterit Lisa 3D Printer to Make Exoskeleton Arms for Children with Spinal Muscular Atrophy – 3DPrint.com

I’ve heard it said that a kid’s main job is just to be a kid – go play, and enjoy the time before…

02.2k

Top 5 Reads for Spinal Muscular Atrophy Patients and Caregivers – SMA News Today

Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that causes a loss of motor neurones and muscle, often causing premature death in…

01.9k

Disabled man who can barely move jailed for armed robbery – Metro

A severely disabled man who can barely move has been jailed for armed robbery in a case that has sparked outrage in Russia. Anton…

01.8k

Disgusted mum blasts ‘scumbags’ who stole disabled daughter’s food bag and pump that she relies on to eat – Mirror.co.uk

An angry mum has lashed out at the “scumbags” who broke into her car and stole her disabled daughter’s food bag. Lisa Warren said…

02.1k

Disney appeal launched for ‘miracle girl’ – Loughborough Echo

THE PARENTS of an eight year old “miracle girl” from East Leake have started an appeal to try and make their little girl’s…

01.8k

First-time mother, 33, shares her anguish as she spends her final months with her terminally ill baby girl – Daily Mail

Like many first time parents, for Rachael and Jonathan the arrival of their first-born child 14 weeks ago was a special and memorable…

01.9k

Nusinersen approved in the EU as first treatment for spinal muscular atrophy – Hospital Healthcare Europe

The European Commission (EC) has granted a marketing authorisation for Spinraza® (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), Biogen has…

01.8k

The cruel disorder killing little Mackenzie – The Courier Mail

THEY can’t cure her. All they can do is spend whatever time they have left with her. And talk about what it is…

02k

Evaluating Olesoxime as a Treatment for Spinal Muscular Atrophy – Medical News Bulletin

While spinal muscular atrophy (SMA) is relatively rare (approximately 1 out of every 11,000 births), there are few treatments currently available to slow…

01.8k

Study opens new line of attack on spinal muscular atrophy – Brown University

Scientists have discovered a physiological chain of events in animal models in which motor neurons and their communication with muscle become disrupted by…

01.9k

New Malden parents raising money to give ‘cheeky and intelligent’ two-year-old with spinal muscular atrophy power chair – Surrey Comet

The parents of a two-year-old boy with spinal muscular atrophy (SMA) are raising money to buy a power chair to give him his…

02.1k

Parents of Northern Ireland toddler with rare muscle-wasting disease say new drug offers hope – Belfast Telegraph

The mother of a little girl who might not live beyond her second birthday has told how access to a new drug has…

01.8k

How motor neurons falter in spinal muscular atrophy – Futurity: Research News

Scientists have discovered a chain of events in which the mutation that causes spinal muscular atrophy disrupts motor neurons and their communication with…

01.8k

Spinal Muscular Atrophy: New Clues to Cause and Treatment – Bioscience Technology

Spinal muscular atrophy (SMA), a neurodegenerative disease that causes progressive muscle wasting and paralysis, may be partly due to abnormalities in the synapses…

01.8k

Spinal muscular atrophy: New clues to cause and treatment – Medical Xpress

Spinal motor neurons (blue) affected by SMA (right image) have significantly less expression…

01.8k

Nusinersen receives positive CHMP opinion for the treatment of spinal muscular atrophy – Hospital Healthcare Europe

Biogen has announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending…

01.5k

Brown University Discovers Cure For Fatal Spinal Muscular Atrophy – University Herald

While spinal muscular atrophy (SMA), generally, has still no cure, experts at Brown University have found a “multipronged counterattack” for older patients and…

01.6k

New line of attack on spinal muscular atrophy – Science Daily

Though spinal muscular atrophy (SMA) in its most severe form remains incurable and fatal in early childhood, researchers are sustaining a multipronged counterattack…

01.4k

Family raising £10000 to help extend baby Ella’s life – LoughboroughEcho.net

Seven-month-old Ella-Rose Long, of Holland Close, Loughborough, who has been diagnosed with…

01.7k

Nusinersen Improves Motor Function in Children with Spinal Muscular Atrophy – Specialty Pharmacy Times

Data from a phase 3 end of study of…

01.4k

Family travels to France for son’s live-saving treatment – ITV News

When I meet Dani Ingrosso and John Else at the Trousseau Hospital in Paris, they are full of energy and positivity. You wouldn’t know…

01.1k

How to Handle Breathing Difficulties in Spinal Muscular Atrophy – SMA News Today

Many children with type 1 and type 2 spinal muscular atrophy (SMA) suffer from breathing difficulties. This is due to the weakened muscles…

01.3k

Spinal Muscular Atrophy Mouse Models Respond to CK-2127107 – SMA News Today

Cytokinetics’ next-generation fast skeletal troponin activator CK-2127107 improves muscle function in mouse models of spinal muscular atrophy (SMA), according…

01.2k

Spinal Muscular Atrophy Symptoms – SMA News Today

Although there are different types of SMA according to the age of onset and severity, SMA affects most all physical movement including walking,…

01.2k

Children with Spinal Muscular Atrophy Have Poor Bone Health, Study Finds – SMA News Today

Children with spinal muscular atrophy (SMA) have poor bone health that leads to common thigh-bone fractures, regardless of the state of their disease,…

01.2k

Global Industry Analysis on Spinal Muscular Atrophy (SMA) Treatment Market, 2015 – 2021 – Medgadget.com (blog)

Logo288 Spinal muscular atrophy (SMA) refers to a group of inherited diseases that affects the functioning of muscles because of deterioration. It typically…

01.4k

Girl fighting spinal muscular atrophy has a dream fishing trip – Sun Sentinel

He was fishing with Madison Smith, a 9-year-old who has spinal muscular atrophy, a disease that has had the little girl in a…

01.2k

Regular Exercise Seen to Protect Motor Neurons in Spinal Muscular Atrophy Animal Study – SMA News Today

SMA currently has no cure and while physical exercise might represent a promising approach to alleviate SMA symptoms, the lack of data on…

01.1k

Long-term exercise may benefit Spinal Muscular Atrophy patients – News-Medical.net

Long-term exercise appears to be beneficial for Spinal Muscular Atrophy (SMA) like mice, suggesting a potential of active physiotherapy for patient care; according…

01k

Spinal muscular atrophy cause uncovered by stem cell research – Bel Marra Health

Spinal muscular atrophy cause uncovered by stem cell research The cause of spinal muscular atrophy, a condition that leads to muscle weakness, has…

01.1k

Exercise may protect nerve cells in Spinal Muscular Atrophy patients – EurekAlert (press release)

Long-term exercise appears to be beneficial for Spinal Muscular Atrophy (SMA) like mice, suggesting a potential of active physiotherapy for patient care; according…

01.1k

Spinal Muscular Atrophy Patients, Caregivers Needed for Medical Research Survey – SMA News Today

Medical education researcher Gregory Salinas, Ph.D. is conducting a survey to learn more about the challenges of patients suffering from type II or…

01.1k

Blocking a Spinal Muscular Atrophy Enzyme, JNK3, Seen to Ease Disease in Mice Regardless of Underlying Mutation – SMA News Today

Texas Tech University researchers have identified a possible therapeutic target, the enzyme JNK3, for spinal muscular atrophy (SMA) that is independent of the…

01.2k

Spinal Muscular Atrophy: What You Need To Know – BioNews Texas

Spinal muscular atrophy (SMA) is a genetic pathology that attacks nerve cells (motor neurons) in the spinal cord. These are the cells that…

01.1k

Higher education briefs: Professor awarded grant for spinal muscular atrophy research – The Herald-Times (subscription)

Elliot J. Androphy, a professor and chairman of the department of dermatology at the Indiana University School of Medicine, has been awarded a…

01.1k

15 SMA Patients to Test AveXis’ Gene Therapy Candidate in Phase I Clinical Trial – SMA News Today

AveXis, Inc., announced that its Phase 1 clinical trial of its proprietary gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy…

01.1k

Artist with spinal muscular atrophy brings exhibition to Wofford – Spartanburg Herald Journal

“The third is someone with spinal muscular atrophy, which is the disease I have,” she adds. “I like using threes because my brother,…

01.2k

Dr. Elliot J. Androphy receives IGNITE grant to develop drug candidate for spinal muscular atrophy – News-Medical.net

Dr. Elliot J. Androphy, the Kampen-Norins Professor and chair of the Department of Dermatology at the Indiana University School of Medicine, has been…

01.1k

Study unveils new therapeutic target for spinal muscular atrophy Texas Tech University Health Sciences Center El Paso – EurekAlert (press release)

Spinal muscular atrophy is a debilitating disease that causes weakness and wasting of the muscles. The disease ranges in severity with patients experiencing…

01.2k

Accomable: Could this Airbnb-style service revolutionize disability travel? – CNN International

(CNN) Srin Madipalli has spinal muscular atrophy — a genetic disorder that affects the control of muscle movement and has confined him to…

01.2k

IU School of Medicine researcher awarded NIH IGNITE grant to develop drug candidate – EurekAlert (press release)

The funding will allow him and his colleagues to test compounds to determine the best candidate to treat spinal muscular atrophy, a genetic…

01.1k

Spinal Muscular Atrophy (SMA) Treatment Market(2015 – 2021): Global Market Study And Analysis. – Medgadget.com (blog)

Logo78 Spinal muscular atrophy (SMA) refers to a group of inherited diseases that affects the functioning of muscles because of deterioration. It typically…

01.1k

Melbourne girl shares her experience of spinal muscular atrophy for hospital appeal awareness – ABC Online

Six-year-old Simone Lionetti has been a regular patient at Melbourne’s Royal Children’s Hospital for most of her life. She was diagnosed with the…

01.1k

Accomable: Could this Airbnb-style service revolutionize disability travel? – CNN

(CNN) Srin Madipalli has spinal muscular atrophy — a genetic disorder that affects the control of muscle movement and has confined him to…

0943

VIDEO: Brooke’s tribute to her cousin Harry – Stratford upon Avon Herald

He had been doing really well but then we had the news that he had spinal muscular atrophy and only had a couple…

0965

Plano resident crowned 2016 Ms. Wheelchair Texas – Star Local Media

Spoonemore’s spinal muscular atrophy has not stopped her from attending college at Baylor University, receiving her degree in computer information systems and entering…

01k

Spinal Muscular Atrophy (SMA) Pipeline Review, Industry Analysis, Market Size and Forecast 2016 – 2020 – Medgadget.com (blog)

“Spinal Muscular Atrophy (SMA) Global 2015 Clinical Trials Review, H2” provides an detailed overview of Spinal Muscular Atrophy (SMA) scenario. Report includes top…

01.1k

Good Friday Appeal poster girl Simone Lionetti doesn’t let her spinal muscular atrophy get her down – Herald Sun

Despite her spinal muscular atrophy Simone has wasted no time getting the most out of life.. Picture: David Caird. The experts had seen…

01.1k

Derby County fan Craig Buckby mysteriously found dead in wheelchair yards from home after Manchester United match – Derby Telegraph

Craig Buckby suffered from spinal muscular atrophy – a genetic condition which can affect up to 2,500 people in the UK each year….

0888

Biomarkers for Spinal Muscular Atrophy Progression Identified – SMA News Today

Early diagnosis of SMA can greatly influence disease treatment, but it is difficult to assess treatment results in clinical trials involving infants mainly…

01.2k

Families affected by muscular dystrophy remind America to ‘Live Unlimited’ – PRWeek

When I was diagnosed with spinal muscular atrophy at 15 months, doctors told my parents I would only live to be 12 years…

0995

MY DISABILITY WON’T STOP ME FROM BEING A MOM – Aljazeera.com

Valeria was 17 months old when she was diagnosed with Spinal Muscular Atrophy (SMA), a genetic disease affecting the nervous system that controls…

0999

Senate Committee Approves 7 Bills in 21st Century Cures Act, More Votes Awaited – SMA News Today

Cure SMA, a nonprofit organization funding research into new treatments and a possible cure for spinal muscular atrophy (SMA), and providing SMA families…

01k

Only biotech companies have braved IPO market this year — with insider help – MarketWatch

AveXis, for example, said it would be using the funds for a clinical trial and future spinal muscular atrophy trials. And it seems…

0932

Limavady man’s appeal to politicians – Derry Journal

A Limavady wheelchair user has made a heartfelt plea to politicians to do more to help people living with disabilities. Raymond Tracey, aged…

01.1k

Valery Spiridonov set to undergo head transplant sells souvenirs to raise funds – Daily Mail

Valery has the potentially fatal Werdnig-Hoffman disease, also known as spinal muscular atrophy, and has been physically handicapped since childhood. He needs to…

01.3k

4-year-old girl in need of treatment for spinal muscular atrophy – KPIC News

KPIC News ROSEBURG, Ore. – Calypso, 4-year-old girl was born with a progressive neuromuscular condition, has a treatment available, but her mother says it’s…

0812

Target Enrollment in Child Spinal Muscular Atrophy Study Earns Ionis $2M Payment From Biogen – SMA News Today

Ionis Pharmaceuticals announced it has completed its target enrollment of the Phase 3 CHERISH study, a trial designed to support marketing approval of…

01.3k

Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development – PR Newswire (press release)

PTC Therapeutics, Inc. (Nasdaq: PTCT) today announced that RG7916, an additional SMN2 splicing modifier from the company’s joint development program with Roche and…

01.3k

Spinal Muscular Atrophy (SMA) Treatment Market (2015 – 2021): Global Market Study And Analysis – Medgadget.com (blog)

Spinal muscular atrophy (SMA) refers to a group of inherited diseases that affects the functioning of muscles because of deterioration. It typically results…

01.3k

We are normal human beings, says Ben, who has spinal muscular atrophy – Swindon Advertiser

Ben Morris has had spinal muscular atrophy all his life. But the 15-year-old, from Middleleaze, refuses to let it get the better of…

01.3k
Font Resize