Spinal Muscular Atrophy – Page 6

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Tel Aviv University Awards Honorary Doctorate to Researcher Who Played Critical Role in Developing Spinal Muscular Atrophy Drug

Post author:James Moore
Post published:May 20, 2019
Post category:Spinal Muscular Atrophy

According to a story from The Jerusalem Post, Adrian Krainer is a prominent molecular geneticist and biochemist. His research was also pivotal in developing the first-ever disease modifying treatment for…

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Evidence Suggests The Spinal Muscular Atrophy Gene Therapy Could Help all Patients Regardless of Severity

Post author:James Moore
Post published:May 9, 2019
Post category:Spinal Muscular Atrophy

According to a story from BNN Bloomberg, data from three studies suggest that Zolgensma, a gene therapy for spinal muscular atrophy developed by Novartis, could be useful to all patients…

Spinraza for Spinal Muscular Atrophy has Finally been made Available in China

Post author:Trudy Horsting
Post published:May 6, 2019
Post category:Spinal Muscular Atrophy

In February, Li Kegiang, Premier of the State Council, announced that the Chinese government was going to take steps to improve rare disease patients access to medicine. As part of…

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Baby’s Death in Spinal Muscular Atrophy Gene Therapy Trial Could be Linked to Treatment, Company Says

Post author:James Moore
Post published:May 3, 2019
Post category:Spinal Muscular Atrophy

According to a story from BNN Bloomberg, Novartis AG, a drug company that is nearing the completion of the development of a potentially groundbreaking new gene therapy for spinal muscular…

Rare Diseases: Only 10% of India’s 1.37 Billion People Have Health Insurance

Post author:Rose Duesterwald
Post published:May 1, 2019
Post category:Duchenne Muscular Dystrophy Gaucher Disease Pompe Disease Spinal Muscular Atrophy

In about five years India’s population will exceed that of China which is currently ranked number one according to population. Contrast these numbers with the fact that only ten percent…

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Recent Study Indicates That SPINRAZA can Benefit Patients with Late-Onset Spinal Muscular Atrophy

Post author:James Moore
Post published:May 1, 2019
Post category:Spinal Muscular Atrophy

According to a story from globenewswire.com, the biotechnology company Biogen recently released data from an open label study that was testing the company's spinal muscular atrophy drug nusinersen (marketed as…

New Drug Under Review by the FDA to Treat the Most Lethal Form of Spinal Muscular Atrophy

Post author:Rose Duesterwald
Post published:April 30, 2019
Post category:Spinal Muscular Atrophy Spinal muscular atrophy type I with congenital bone fractures

According to an article in Biospace, the FDA is currently reviewing data from the Phase III STRIVE clinical trial for possible approval of Zolgensma as gene therapy for spinal…

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More Evidence Suggesting Nusinersen Effective for Spinal Muscular Atrophy Types II and III

Post author:Scott Carlson
Post published:April 29, 2019
Post category:Spinal Muscular Atrophy

According to a press release from the American Academy of Neurology published by EurekAlert, new research published in Neurology further suggests that established spinal muscular atrophy (SMA) drug nusinersen could…

courtesy of Anna Landre

Patient Spotlight: Anna Landre

Post author:Scott Carlson
Post published:April 25, 2019
Post category:Spinal Muscular Atrophy

Anna Landre is a sophomore at Georgetown University in Washington, D.C. It’s a good school, you’ve probably heard of it. On top of the normal stresses of her college life,…

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Latest Data Reinforces Effectiveness of a Potential Gene Therapy for Type 1 Spinal Muscular Atrophy

Post author:James Moore
Post published:April 23, 2019
Post category:Spinal Muscular Atrophy

According to a story from inpublic.globenewswire.com, the Novartis company AveXis has released interim data from an ongoing Phase 3 clinical trial of Zolgensma, a gene therapy drug that is being…

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Report Calls for Reducing Prices on Spinal Muscular Atrophy Drugs

Post author:James Moore
Post published:April 16, 2019
Post category:Rare Disease Spinal Muscular Atrophy

According to a story from Xconomy, a recent report from the Institute for Clinical and Economic Review (ICER), based on Boston, is calling for reductions to the price tags of…

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NZ Grandmas Push for Spinal Muscular Atrophy Drug Coverage

Post author:James Moore
Post published:April 12, 2019
Post category:Rare Disease Spinal Muscular Atrophy

According to a story from Radio NZ, two grandmothers have taken a central role in getting a petition delivered to the parliament of New Zealand. This petition is calling for…

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Gene Therapy Could Change Rare Disease Treatment Forever, But Will Anyone be Able to Afford It?

Post author:James Moore
Post published:April 12, 2019
Post category:Rare Disease Spinal Muscular Atrophy

According to a story from BNN Bloomberg, gene therapies are without a doubt an upcoming advancement in care and treatment for many diseases that have a genetic origin. This includes…

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Study Indicates Safety of New Potential Treatment for Swallowing Dysfunction in Kennedy Disease

Post author:Trudy Horsting
Post published:March 22, 2019
Post category:Kennedy disease Spinal Muscular Atrophy

Kennedy disease or, Spinal and Bulbar Muscular Atrophy (SBMA) is a form of Spinal Muscular Atrophy (SMA). SBMA causes muscle weakness throughout the limbs which results in impaired mobility (typical…

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Bioscience Companies in Ohio Take Aim at Rare Diseases

Post author:James Moore
Post published:March 15, 2019
Post category:Batten Disease Cystic Fibrosis Duchenne Muscular Dystrophy Rare Disease Sanfilippo Syndrome Spinal Muscular Atrophy

According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…

The Major Ethical Dilemma Behind Rare Disease Drug Costs and a Bioethicist’s Solution

Post author:Trudy Horsting
Post published:March 13, 2019
Post category:Batten Disease Spinal Muscular Atrophy

Avraham Steinberg recently spoke at RARE2019, the 2nd International Congress on Advanced Treatments in Rare Diseases. It was held on March 4th and 5th in Vienna, Austria. His talk focused…

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The Barriers to Developing Rare Disease Drugs

Post author:James Moore
Post published:March 8, 2019
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy Rare Disease Spinal Muscular Atrophy

According to a story from Xtalks, there has been an uptick in research related to rare diseases and the development of new therapies for them. Despite this, only a small…

New Treatment for Familial Chylomicronemia Syndrome is Close to Approval in the EU

Post author:Trudy Horsting
Post published:March 7, 2019
Post category:Amyloidosis Familial Chylomicronemia Syndrome Familial Partial Lipodystrophy Spinal Muscular Atrophy

Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…

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Ireland Lags Behind the Rest of Europe in Approving Rare Disease Drugs

Post author:James Moore
Post published:March 5, 2019
Post category:Rare Disease Spinal Muscular Atrophy

According to a story from The Irish Times, a recent report issued by the Irish Pharmaceutical Healthcare Association (IPHA) indicates that Ireland trails many other European countries in approving new…

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FDA Releases New Info About How it Could Regulate Gene Therapy

Post author:James Moore
Post published:March 4, 2019
Post category:Duchenne Muscular Dystrophy Spinal Muscular Atrophy

According to a story from finance.yahoo.com, the US Food and Drug Administration (FDA) has recently released new guidelines that are related to how the agency will regulate and manage the…

Report Explains How NICE Policies are Failing Rare Disease Patients

Post author:Trudy Horsting
Post published:February 28, 2019
Post category:Rare Disease Spinal Muscular Atrophy

NICE stands for the National Institute for Health and Care Excellence in England. This organization is responsible for evaluating the cost effectiveness of all medications as they come to the…

Florida 17th State to Add Spinal Muscular Atrophy to Their Newborn Screening Panel

Post author:Trudy Horsting
Post published:February 26, 2019
Post category:Spinal Muscular Atrophy

Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is caused by a mutation in the SMN1 gene. This mutation results in a loss of motor neurons causing weak and atrophied muscles.…

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Scotland to Offer Spinal Muscular Atrophy Drug Spinraza Through NHS

Post author:Scott Carlson
Post published:February 26, 2019
Post category:Spinal Muscular Atrophy

According to a publication from SMA News Today, Scotland is now the latest country to make spinal muscular atrophy (SMA) drug Spinraza available to patients at low or no cost…

RARE2019 Conference to Take Place this Week in Austria

Post author:Trudy Horsting
Post published:February 26, 2019
Post category:Gaucher Disease Spinal Muscular Atrophy Timely

Second Annual International Congress on Advanced Treatments in Rare Diseases The first International Congress on Advanced Treatments in Rare Diseases, otherwise known as RARE2018, was held in London, England. Now,…

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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects

Post author:Trudy Horsting
Post published:February 21, 2019
Post category:Duchenne Muscular Dystrophy Hemophilia Muscular Dystrophy Spinal Muscular Atrophy

Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…

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Tel Aviv University Awards Honorary Doctorate to Researcher Who Played Critical Role in Developing Spinal Muscular Atrophy Drug

Evidence Suggests The Spinal Muscular Atrophy Gene Therapy Could Help all Patients Regardless of Severity

Spinraza for Spinal Muscular Atrophy has Finally been made Available in China

Baby’s Death in Spinal Muscular Atrophy Gene Therapy Trial Could be Linked to Treatment, Company Says

Rare Diseases: Only 10% of India’s 1.37 Billion People Have Health Insurance

Recent Study Indicates That SPINRAZA can Benefit Patients with Late-Onset Spinal Muscular Atrophy

New Drug Under Review by the FDA to Treat the Most Lethal Form of Spinal Muscular Atrophy

More Evidence Suggesting Nusinersen Effective for Spinal Muscular Atrophy Types II and III

Patient Spotlight: Anna Landre

Latest Data Reinforces Effectiveness of a Potential Gene Therapy for Type 1 Spinal Muscular Atrophy

Report Calls for Reducing Prices on Spinal Muscular Atrophy Drugs

NZ Grandmas Push for Spinal Muscular Atrophy Drug Coverage

Gene Therapy Could Change Rare Disease Treatment Forever, But Will Anyone be Able to Afford It?

Study Indicates Safety of New Potential Treatment for Swallowing Dysfunction in Kennedy Disease

Bioscience Companies in Ohio Take Aim at Rare Diseases

The Major Ethical Dilemma Behind Rare Disease Drug Costs and a Bioethicist’s Solution

The Barriers to Developing Rare Disease Drugs

New Treatment for Familial Chylomicronemia Syndrome is Close to Approval in the EU

Ireland Lags Behind the Rest of Europe in Approving Rare Disease Drugs

FDA Releases New Info About How it Could Regulate Gene Therapy

Report Explains How NICE Policies are Failing Rare Disease Patients

Florida 17th State to Add Spinal Muscular Atrophy to Their Newborn Screening Panel

Scotland to Offer Spinal Muscular Atrophy Drug Spinraza Through NHS

RARE2019 Conference to Take Place this Week in Austria

Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects

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