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Your search keyword '"Spinal muscular atrophy -- Care and treatment"' showing total 16 results
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16 results on '"Spinal muscular atrophy -- Care and treatment"'

1. Sir Christopher Chope ask the Secretary of State for Health and Social Care, how many sufferers of spinal muscular atrophy have not yet received the drug spinraza for which they are eligible following the decision of NICE; and if he will make a statement

Subjects
Spinal muscular atrophy -- Care and treatment, Health care reform, Business, international, Spinraza (Medication)
Abstract

London: UK Parliament has issued the following written answer: Answered by: Jo Churchill Answered on: 25 June 2020 As at June 2020, the number of patients receiving Spinraza for the [...]

Published
2020

2. New gene therapy to treat spinal muscular atrophy

Subjects
Spinal muscular atrophy -- Care and treatment, Gene therapy, Genes, Muscular atrophy, Marketing, Muscle weakness, Diseases, Business, international, European Union, Zolgensma (Medication)
Abstract

Amsterdam, the Netherlands: European Medicines Agency has issued the following news release: EMA has recommended granting a conditional marketing authorisation in the European Union for the gene therapy Zolgensma (onasemnogene [...]

Published
2020

3. Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma(r) the only gene therapy for patients with spinal muscular atrophy (SMA)

Subjects
Novartis AG, Spinal muscular atrophy -- Care and treatment, Gene therapy, Genes, Pharmaceutical industry, Neurons, Muscular atrophy, Antibodies, Health, Diseases, General interest, News, opinion and commentary, Zolgensma (Medication)
Abstract

Basel: Novartis has issued the following press release: Novartis Pharma K.K. ('Novartis Pharma') today announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) approved Zolgensma(r) (onasemnogene abeparvovec) for [...]

Published
2020

4. UK Contract Notice: NHS Arden and Greater East Midlands Commissioning Support Unit Issues Solicitation for 'Treatment to adult patients who have 5Q Spinal Muscular Atrophy (SMA) with the Drug Nusinersen (2)'

Subjects
Spinal muscular atrophy -- Care and treatment, Nusinersen, Adults, Muscular atrophy, Vendor relations, Contract agreement, Business, international
Abstract

London: NHS Arden and Greater East Midlands Commissioning Support Unit has issued requirement for 'Treatment to adult patients who have 5Q Spinal Muscular Atrophy (SMA) with the Drug Nusinersen (2)' [...]

Published
2020

5. PTC Therapeutics Announces FDA Grants Priority Review to Risdiplam for the Treatment of Spinal Muscular Atrophy

Subjects
United States. Food and Drug Administration, PTC Therapeutics Inc., Spinal muscular atrophy -- Care and treatment, Pharmaceutical industry, Prescriptions (Drugs), Muscular atrophy, Legal fees, General interest, News, opinion and commentary
Abstract

SOUTH PLAINFIELD: PTC Therapeutics, Inc. has issued the following press release: PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the United States Food and Drug Administration (FDA) has granted priority [...]

Published
2019

6. Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy

Subjects
Clinical trials, Spinal muscular atrophy -- Care and treatment, Muscular atrophy, General interest, News, opinion and commentary
Abstract

CAMBRIDGE: Scholar Rock has issued the following press release: -- Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors [...]

Published
2019

7. Novartis stands behind Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy

Subjects
Novartis AG, Spinal muscular atrophy -- Care and treatment, Pharmaceutical industry, Muscular atrophy, Marketing, Children, General interest, News, opinion and commentary, Zolgensma (Medication)
Abstract

Basel: Novartis AG has issued the following press release: Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma(r) (onasemnogene abeparvovec-xioi). First [...]

Published
2019

8. Novartis stands behind Zolgensma(r) (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy

Subjects
United States. Food and Drug Administration, Spinal muscular atrophy -- Care and treatment, Muscular atrophy, Marketing, Children, General interest, News, opinion and commentary, Zolgensma (Medication)
Abstract

Zurich, Switzerland: SIX Swiss Exchange Ltd has issued the following notice: Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma(r) (onasemnogene [...]

Published
2019

9. Sir Christopher Chope ask the Secretary of State for Health and Social Care, pursuant to the Answer of 8 July 2019 to Question 369611 on Spinal Muscular Atrophy (SMA), what the evidential basis is that Spinraza does not work effectively on people with SMA that are excluded from access to that drug following the announcement of 15 May 2019 by NICE on that drug; and if he will make a statement

Subjects
Spinal muscular atrophy -- Care and treatment, Muscular atrophy, Technology, Health, Socialized medicine, Business, international, Spinraza (Medication)
Abstract

London: United Kingdom House of Commons has issued the following written answer: A Answered by: Seema Kennedy Answered on: 15 July 2019 The National Institute for Health and Care Excellence [...]

Published
2019

10. Sir Christopher Chope( ask the Secretary of State for Health and Social Care, pursuant to the Answer of 1 July 2019 to Question 269609 on Spinal Muscular Atrophy, if he will place in the Library a copy of the managed access agreement

Subjects
Spinal muscular atrophy -- Care and treatment, Libraries, Nusinersen, Muscular atrophy, Muscular dystrophy, Biological products industry, Health, Business, international
Abstract

London: United Kingdom House of Commons has issued the following written answer: Answered by: Seema Kennedy Answered on: 08 July 2019 The Managed Access Agreement for nusinersen for the treatment [...]

Published
2019

11. Sir Christopher Chope ask the Secretary of State for Health and Social Care, for what reason the announcement by NICE on 15 May 2019 that it would facilitate access by people with spinal muscular atrophy to a new drug for prescription on the NHS was followed several weeks later by an announcement that the drug would be available only to a specific group of those people; and if he will make a statement

Subjects
Spinal muscular atrophy -- Care and treatment, Nusinersen, Health care reform, Muscular atrophy, Biological products industry, Business, international
Abstract

London: United Kingdom House of Commons has issued the following written answer: Answered by: Seema Kennedy Answered on: 01 July 2019 The National Institute for Health and Care Excellence (NICE) [...]

Published
2019

13. Public summary of opinion on orphan designation Adeno-associated viral vector serotype hu68 containing the human SMN1 gene for the treatment of spinal muscular atrophy

Subjects
Orphans, Spinal muscular atrophy -- Care and treatment, Genes, Muscular atrophy, Business, international
Abstract

Amsterdam: European Medicines Agency has issued the following document: source is acknowledged. 22 November 2018 EMA/530909/2018 Public summary of opinion on orphan designation Adeno-associated viral vector serotype hu68 containing the [...]

Published
2019

14. BIOGEN'S SPINRAZA(r)V (NUSINERSEN) RECEIVES RECOMMENDATION FROM NICE FOR THE TREATMENT OF INFANTS, CHILDREN AND ADULTS WITH 5q SPINAL MUSCULAR ATROPHY

Subjects
Nusinersen, Spinal muscular atrophy -- Care and treatment, Infants, Biological products industry, Socialized medicine, Vice presidents (Organizations), Muscular atrophy, General interest, News, opinion and commentary, Spinraza (Medication)
Abstract

MAIDENHEAD, England: Biogen has issued the following press release: The National Institute for Health and Care Excellence (NICE) has recommended funding for Spinraza (nusinersen) on the National Health Service (NHS). [...]

Published
2019

15. Mr Adrian Bailey ask the Secretary of State for Health and Social Care, what progress has been made on concluding a managed access treatment agreement for the use of Spinraza for patients with spinal muscular atrophy types 2 and 3

Subjects
Nusinersen, Spinal muscular atrophy -- Care and treatment, Biological products industry, Health, Muscular atrophy, Business, international, Spinraza (Medication)
Abstract

London: United Kingdom House of Commons has issued the following written answer: Answered by: Steve Brine Answered on: 28 February 2019 The National Institute for Health and Care Excellence (NICE) [...]

Published
2019

16. AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1

Subjects
United States. Food and Drug Administration, Spinal muscular atrophy -- Care and treatment, Genes, Pediatrics, General interest, News, opinion and commentary
Abstract

CHICAGO: AveXis, Inc. has issued the following news release: AveXis, Inc., (Nasdaq:AVXS) a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today [...]

Published
2016

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