'Unthinkable' that the NHS won't buy life-saving children's drug
Doctors have said it is 'unthinkable' that British children can't get access to the £450,000-a-year drug Spinraza, which can improve the crippling condition spinal muscular atrophy. (Source: the Mail online | Health)
Source: the Mail online | Health - October 18, 2018 Category: Consumer Health News Source Type: news

Breakthrough Prizes Recognize Aneuploidy Researcher, Biochemist
This year's winners also include the developers of nusinersen, an oligonucleotide therapeutic for spinal muscular atrophy. (Source: The Scientist)
Source: The Scientist - October 18, 2018 Category: Science Tags: News & Opinion Source Type: news

URMC Joins Network Dedicated to Improving Care for Spinal Muscular Atrophy
The University of Rochester Medical Center has been selected as one of the first four institutions in the U.S. to participate in the SMA Care Center Network. The network is being created as new treatments and approaches to care are transforming how spinal muscular atrophy is treated. (Source: University of Rochester Medical Center Press Releases)
Source: University of Rochester Medical Center Press Releases - October 15, 2018 Category: Universities & Medical Training Authors: University of Rochester Medical Center Source Type: news

Roche announces new data for risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress
Roche today announced interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in SMA. (Source: Roche Media News)
Source: Roche Media News - October 3, 2018 Category: Pharmaceuticals Source Type: news

Roche announces new data for risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress
Roche today announced interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in SMA. (Source: Roche Investor Update)
Source: Roche Investor Update - October 3, 2018 Category: Pharmaceuticals Source Type: news

New Delivery System for Nusinersen Safe in SMA New Delivery System for Nusinersen Safe in SMA
A new subcutaneous intrathecal catheter system may be a safe and relatively well-tolerated alternative to lumbar puncture for delivering nusinersen to adults and children with spinal muscular atrophy.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - September 25, 2018 Category: Consumer Health News Tags: Pediatrics News Source Type: news

Insuring patient access and affordability for treatments for rare and ultra-rare diseases
(American College of Medical Genetics and Genomics) The last decade has seen tremendous progress in the development of new drugs for patients with genetic disorders, including Cystic Fibrosis, many lysosomal storage disorders (Gaucher disease, Fabry disease and others) and most recently, Duchenne Muscular Dystrophy and Spinal Muscular Atrophy (SMA). The American College of Medical Genetics and Genomics (ACMG) is concerned with the staggering projected cost of these new treatments. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 7, 2018 Category: International Medicine & Public Health Source Type: news

Researchers correlate spinal muscular atrophy disease expression with haplotypes
(Clinic for Special Children) A natural history study has provided the first comprehensive clinical description of spinal muscular atrophy (SMA) within the Amish and Mennonite communities and correlates ancestral chromosome 5 haplotypes and SMN2 copy number with disease severity. SMA is a devastating genetic disease that affects the motor neurons that control movement, eating, and breathing. The observations were conducted within a population-specific framework to elucidate subtle differences in disease expression and the subsequent impact of disease-modifying therapies administered early in life. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 6, 2018 Category: International Medicine & Public Health Source Type: news

Hospital costs high in children with spinal muscular atrophy  1
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - September 1, 2018 Category: Drugs & Pharmacology Source Type: news

Nusinersen (Spinraza) for Spinal Muscular Atrophy May Help Older Children: Study
WEDNESDAY, Aug. 29, 2018 -- A drug used to treat a rare and deadly muscle-wasting disease in children still improves muscle control even if treatment begins at a later age, a new study found. Spinal muscular atrophy (SMA) is a leading genetic cause... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - August 29, 2018 Category: General Medicine Source Type: news

New nusinersen drug delivery method identified for spinal muscular atrophy patients
(Clinic for Special Children) A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy (SMA) using a subcutaneous intrathecal catheter system (SIC) configured by connecting an intrathecal catheter to an implantable infusion port. SMA is a devastating genetic disease that leads to progressive degeneration of motor neurons that control movement, swallowing, and breathing. It is the leading genetic cause of infant death worldwide. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 24, 2018 Category: International Medicine & Public Health Source Type: news

NICE welcomes further discussions about spinal muscular atrophy drug
NICE hopes for further talks over nusinersen for treating a rare genetic disorder; spinal muscular atrophy (SMA). (Source: NHS Networks)
Source: NHS Networks - August 14, 2018 Category: UK Health Source Type: news

Dr. Jekyll, Mr. Hyde: Study reveals healing mesenchymal cells morph and destroy muscles in models of spinal cord injury, ALS and spinal muscular atrophy
(Sanford Burnham Prebys Medical Discovery Institute) Scientists at Sanford Burnham Prebys Medical Discovery Institute (SBP), in collaboration with the Fondazione Santa Lucia IRCCS in Rome, have discovered a new disease-specific role in FAP cells in the development of muscle tissue wasting, indicating a potential new avenue for treating motor neuron diseases including spinal cord injury, ALS and spinal muscular atrophy. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - July 26, 2018 Category: Biology Source Type: news

KC startup lands funding to research treatment of rare infant disease
A Kansas City-based pharmaceutical startup landed $3 million in funding to study a treatment for spinal muscular atrophy.   A rare genetic disease, spinal muscular atrophy, leads to weak muscles, trouble breathing and swallowing. Infants diagnosed with SMA type I, the most severe and most common form of the disease, can't lift their heads. Nearly 80 percent don't live to age 2. "It's terrible," Shift Pharmaceutical s CEO Steve O'Connor told the Kansas City Business Journal. "Basically, these kids… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - July 24, 2018 Category: American Health Authors: Elise Reuter Source Type: news

KC startup lands funding to research treatment of rare infant disease
A Kansas City-based pharmaceutical startup landed $3 million in funding to study a treatment for spinal muscular atrophy.   A rare genetic disease, spinal muscular atrophy, leads to weak muscles, trouble breathing and swallowing. Infants diagnosed with SMA type I, the most severe and most common form of the disease, can't lift their heads. Nearly 80 percent don't live to age 2. "It's terrible," Shift Pharmaceutical s CEO Steve O'Connor told the Kansas City Business Journal. "Basically, these kids… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - July 24, 2018 Category: Biotechnology Authors: Elise Reuter Source Type: news

Biogen quarterly sales rise 9 percent
Biogen Inc reported a 9 percent rise in quarterly revenue on Tuesday, helped by demand for its spinal muscular atrophy drug Spinraza. (Source: Reuters: Health)
Source: Reuters: Health - July 24, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Early treatment with nusinersen can mean better outcomes for babies
(IOS Press) Spinal muscular atrophy (SMA) is a genetic disease that affects motor neurons in the spinal cord, resulting in muscle atrophy and widespread weakness that eventually impair swallowing and breathing. A new study in the Journal of Neuromuscular Diseases finds that children with SMA type 1 can achieve improvements in motor function after six months of treatment with the drug nusinersen, particularly when treatment began before seven months of age. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 16, 2018 Category: International Medicine & Public Health Source Type: news

Roche SMA treatment scores positive trial results
Roche reported glowing results from a trial for a new treatment for spinal muscular atrophy (SMA). (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - June 18, 2018 Category: Pharmaceuticals Source Type: news

Roche SMA drug shines in study as costly new therapies advance
ZURICH (Reuters) - A drug being co-developed by Roche to treat spinal muscular atrophy (SMA) helped improve development scores in babies with the genetic disease, a study released on Monday showed, as the race heats up for therapies destined to be among the drug industry's most expensive. (Source: Reuters: Health)
Source: Reuters: Health - June 18, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Leg exercise is critical to brain and nervous system health
(Frontiers) New research shows that using the legs, particularly in weight-bearing exercise, sends signals to the brain that are vital for the production of healthy neural cells. The groundbreaking study fundamentally alters brain and nervous system medicine -- giving doctors new clues as to why patients with motor neuron disease, multiple sclerosis, spinal muscular atrophy and other neurological diseases often rapidly decline when their movement becomes limited. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - May 23, 2018 Category: International Medicine & Public Health Source Type: news

Scientists investigate new strategy to treat spinal muscular atrophy in infants
(Scripps Research Institute) Scientists investigate new strategy to treat spinal muscular atrophy in infants. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 8, 2018 Category: International Medicine & Public Health Source Type: news

Nusinersen listed for spinal muscular atrophy in Australia
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - May 1, 2018 Category: Drugs & Pharmacology Source Type: news

Oral Drug Increases SMN Protein in Spinal Muscular Atrophy Oral Drug Increases SMN Protein in Spinal Muscular Atrophy
Preliminary results of the FIREFISH study show a 6.5-fold increase in a protein that's diminished in babies with spinal muscular atrophy.Medscape Medical News (Source: Medscape Critical Care Headlines)
Source: Medscape Critical Care Headlines - April 23, 2018 Category: Intensive Care Tags: Neurology & Neurosurgery News Source Type: news

Biogen To Pay Ionis $1 Billion To Develop Brain Drugs
Their first collaboration, Spinraza, a treatment for a deadly rare disease called spinal muscular atrophy, generated $884 million sales last year, its first on the market. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - April 20, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:BIIB NASDAQ:IONS Source Type: news

AAN: New Drug May Help Infants With Spinal Muscular Atrophy
THURSDAY, April 19, 2018 -- For infants with type 1 spinal muscular atrophy who have two copies of the survival motor neuron (SMN) 2 gene, RG7916 can increase SMN protein levels in the blood, according to a study presented at the annual meeting of... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 19, 2018 Category: Pharmaceuticals Source Type: news

New advances in medication for muscle disease in children
(University of Gothenburg) Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of neuromuscular disease, spinal muscular atrophy (SMA), which afflicts children from 6 to 18 months of age. That is shown by a study published in the New England Journal of Medicine (NEJM). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 26, 2018 Category: International Medicine & Public Health Source Type: news

New drug improves motor function of children with genetic disorder
(Nemours) Children with later-onset spinal muscular atrophy (SMA) were more likely to show gains in motor function when treated with a new medication compared to children receiving a sham procedure, according to a study published today in the New England Journal of Medicine. The study demonstrates the impact the drug, nusinersen, can have on older patients with this progressive neuromuscular disorder. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 14, 2018 Category: International Medicine & Public Health Source Type: news

CIHR grants to the tune of $1.5 million for Professors Descoteaux and Patten
(Institut national de la recherche scientifique - INRS) Following their latest grant competition, the Canadian Institutes of Health Research announced a total of $1.5 in funding for two INRS professors. Professor Albert Descoteaux will receive $918,000 over five years to study the parasite Leishmania, specifically the mechanisms it uses to evade our immune system. Professor Kessen Patten will receive $627,300 over five years as well to enhance the understanding of the mechanisms involved in spinal muscular atrophy, a fatal and untreatable illness that is still poorly understood. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 14, 2018 Category: International Medicine & Public Health Source Type: news

George Lockley is being slowly crippled by SMA
George Lockley suffers from Spinal Muscular Atrophy (SMA), yet doctors believe the way UK medicines are approved could see him miss out on treatment. His parents tell his story. (Source: the Mail online | Health)
Source: the Mail online | Health - February 12, 2018 Category: Consumer Health News Source Type: news

New treatment for SMA offers hope for Arianna
For the first few months of Arianna Condon’s life, everything was moving along fine. She was a happy baby, and seemed to be developing much like her older sister, Tessa. “She was gaining weight, and seemed to be doing great,” says Arianna’s mom, Marina. “She did have problems with reflux, but it was nothing too unusual for a baby.” But by the time Arianna was 3 months old, Marina started to have concerns. Arianna wasn’t lifting her head the way Tessa had at that age. Something didn’t seem right. “I brought it up to her pediatrician, but she told me that all babies devel...
Source: Thrive, Children's Hospital Boston - January 16, 2018 Category: Pediatrics Authors: Ellen Greenlaw Tags: Diseases & Conditions Our Patients’ Stories Dr. Basil Darras spinal muscular atrophy spinal muscular atrophy program Spinraza Source Type: news

Plotting its first drug trial, Cambridge's Scholar Rock bags another $47M
The last we heard from Scholar Rock Inc., the Cambridge startup was unveiling plans in June to develop a drug for spinal muscular atrophy — the same rare infant disease for which Biogen scored a landmark FDA approval in December 2016. On Wednesday, the 45-employee company announced a significant new round of financing that its CEO described in no less ambitious terms: "The primary focus here is to build a blue-chip company," Nages h Mahanthappa said after closing the $47 million Series C financing. The… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 3, 2018 Category: Biotechnology Authors: Max Stendahl Source Type: news

This may be a turning point in treating neurodegenerative diseases
Success in trials for Huntington ’s and Spinal Muscular Atrophy raises hopes that diseases such as Alzheimer’s and ALS could be tackled using a new class of drugsThey are diseases that threaten more than physical health: memories, personality, and the ability to move and speak are incrementally stolen. And until this year neurodegenerative diseases, from Alzheimer ’s toALS, had been entirely unstoppable.However, abreakthrough in Huntington ’s disease this week suggests this bleak picture could be about to change. The landmark trial was the first to show that the genetic defect that causes Huntington...
Source: Guardian Unlimited Science - December 15, 2017 Category: Science Authors: Hannah Devlin Science correspondent Tags: Medical research Neuroscience Alzheimer's Motor neurone disease Parkinson's disease Genetics Drugs Health Biology Source Type: news

Most people in favor of screening for spinal muscular atrophy
(University of Warwick) Research from the University of Warwick indicates that most people are in the UK are in favor of newborn screening for the potentially deadly condition spinal muscular atrophy (SMA). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 5, 2017 Category: International Medicine & Public Health Source Type: news

These 12 People Are Changing Science
On Sunday, some of the smartest minds in science and math will gather at the NASA Ames Research Center in Silicon Valley for the Breakthrough Prize, one of the biggest prizes in science. Several scientists will be recognized for their research, with 12 individuals receiving substantial funding for their work in advancing life sciences, physics and mathematics. Founded in 2012 by Sergey Brin, Mark Zuckerberg & Priscilla Chan, Yuri & Julia Milner, and Anne Wojcicki, this year the annual prize provided $22 million in awards. Here are some of the winners whose work may be changing your life soon: PHYSICS Wilkinson Mi...
Source: TIME.com: Top Science and Health Stories - December 4, 2017 Category: Consumer Health News Authors: Alice Park, Alexandra Sifferlin and Jeffrey Kluger Tags: Uncategorized anne wojcicki Breakthrough Prize healthytime Mark Zuckerberg Math onetime Physics priscilla chan Science sergey brin Silicon Valley Yuri Milner Source Type: news

Gene Therapy, New Drug Battle a Rare but Deadly Disease in Kids
Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Spinal Muscular Atrophy (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - November 2, 2017 Category: Consumer Health News Source Type: news

Gene Therapy, New Drug Fight Rare Disease in Kids
Two innovative new therapies for spinal muscular atrophy (SMA) type 1 have proven highly effective in clinical trials, researchers report. (Source: WebMD Health)
Source: WebMD Health - November 1, 2017 Category: Consumer Health News Source Type: news

Gene Therapy, Antisense Show Gains in Spinal Muscular Atrophy Gene Therapy, Antisense Show Gains in Spinal Muscular Atrophy
An antisense oligonucleotide drug and gene therapy each extended survival and led to attainment of motor milestones in some children with spinal muscular atrophy.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - November 1, 2017 Category: Consumer Health News Tags: Pediatrics News Source Type: news

Gene Therapy, New Drug, Spinraza, Battle Rare Spinal Muscular Atrophy in Kids
WEDNESDAY, Nov. 1, 2017 -- Babies born with a previously untreatable degenerative nerve disease now have two fresh sources of hope for their future. Two innovative new therapies for spinal muscular atrophy (SMA) type 1 have proven highly effective... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - November 1, 2017 Category: General Medicine Source Type: news

New drug enables infants with genetic disorder to live longer, gain motor function
(Nemours) Infants with the most severe form of spinal muscular atrophy (SMA) were more likely to show gains in motor function and were 47 percent more likely to survive without permanent assisted ventilation support when treated with a new medication, according to a study published today in the New England Journal of Medicine. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - November 1, 2017 Category: Biology Source Type: news

G-CON Manufacturing Announces Completion and Delivery of G-CON...
PODs provide the environments for the entire upstream and downstream process for AveXis’ initial product candidate, AVXS-101, a treatment in development for spinal muscular atrophy (SMA) Type 1, the...(PRWeb October 23, 2017)Read the full story at http://www.prweb.com/releases/2017/10/prweb14830297.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - October 23, 2017 Category: Pharmaceuticals Source Type: news

Biogen raises 2017 forecast on Spinraza success
(Reuters) - Biogen Inc's second-quarter profit smashed estimates on Tuesday, boosted by strong sales of its spinal muscular atrophy (SMA) drug, Spinraza, which is priced at $750,000 for the first year of therapy. (Source: Reuters: Health)
Source: Reuters: Health - July 25, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Mother anguish terminally ill newborn baby dying months
Rachael and Jonathan, from Sydney, were over the moon to become first-time parents, until they found out their baby girl, Mackenzie, had Spinal Muscular Atrophy. Rachael spoke to FEMAIL. (Source: the Mail online | Health)
Source: the Mail online | Health - June 22, 2017 Category: Consumer Health News Source Type: news

European Commission approves marketing authorisation for Biogen ’s 5q SMA treatment
The European Commission (EC) has granted marketing authorisation for US-based biotechnology company Biogen ’s Spinraza (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA). (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - June 1, 2017 Category: Pharmaceuticals Source Type: news

EC Approves First Treatment for SMA: Nusinersen (Spinraza) EC Approves First Treatment for SMA: Nusinersen (Spinraza)
The European Commission has granted marketing authorization for the first treatment for 5q spinal muscular atrophy in the European Union.International Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - June 1, 2017 Category: Consumer Health News Tags: Neurology & Neurosurgery News Alert Source Type: news

SPINRAZA(R) (nusinersen) Approved in the European Union
First Approved Treatment in EU for SMA Approved to Treat Broad Range of People with SMA Ionis Earns $50 Million Milestone CARLSBAD, Calif., June 1, 2017 -- (Healthcare Sales & Marketing Network) -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) annou... Biopharmaceuticals, Regulatory Ionis Pharmaceuticals, SPINRAZA, nusinersen, spinal muscular atrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 1, 2017 Category: Pharmaceuticals Source Type: news

Biogen rare infant disease drug Spinraza approved in E.U.
Back in December, Biogen ’s Spinraza became the first drug approved by the FDA to treat spinal muscular atrophy, a top genetic cause of death in infants. Now, the Cambridge-based company has repeated that feat in Europe. Biogen (Nasdaq: BIIB) said Thursday that the European Commission has approved Spinraza, meaning it wi ll become the first drug available in the European Union for SMA patients. The disease is characterized by progressive muscle weakness, and is often fatal. The launch of Spinraza… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - June 1, 2017 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

EU approves Biogen's Spinraza for lead genetic cause of infant death
(Reuters) - Biogen Inc on Thursday said its drug, Spinraza, had secured European Union approval to treat spinal muscular atrophy (SMA), a leading genetic cause of death in infants. (Source: Reuters: Health)
Source: Reuters: Health - June 1, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Cytokinetics receives FDA orphan drug status for Ck-2127107 to treat SMA
Biopharmaceutical company Cytokinetics ’ CK-2127107 has received orphan drug designation from the Office of Orphan Products Development of the US Food and Drug Administration (FDA) for the potential treatment of spinal muscular atrophy (SMA). (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - May 16, 2017 Category: Pharmaceuticals Source Type: news

Neighborhood Health Plan changes course, will cover Biogen drug for all patients
Citing new clinical data presented last week by Cambridge drugmaker Biogen, the health insurance plan run by Partners HealthCare says it ’s changing its policy and will now pay for a newly approved drug intended to treat all patients with the rare genetic disease, spinal muscular atrophy. In an email to the Boston Business Journal today, Neighborhood Health Plan Chief Medical Officer Dr. Anton Dodek confirmed that the company deci ded to pay for the drug, called Spinraza, for all patients with SMA. “Based… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - May 2, 2017 Category: American Health Authors: Don Seiffert Source Type: news