MadDispose of you, February 28, 2019 – Combined with World Day of rare diseases, a & # 39; medicine company Biogen Inc. to identify a & # 39; The first year in the Spanish market by nusinersen, the first and only cure agreed in the world for the treatment of Spinal Muscular Atrophy (AME)1 rare disease of clinical experts who estimate that the number of Patient lives 300 to 350.2
In the last decade, Biogen has been one of the pharmaceutical companies that have researched and developed catastrophic cures in a variety of stupid and intense diseases. So, this company has been a "#; The world's first pharmacy for medical marketing (nusinersen) for the treatment of a patient with SMA.1 This milestone has changed the natural nature of its philosophy and has made progress on the progress of many patients. This happened just a few years ago when the head of the Ministry of Health, Social Services and Equality informed the Foundation Atrophia Muscular Espinal (FundAME) Foundation of this great step to change the history of its disease. Spain was going to be one of the first countries to take this drug into medical services.3
Currently, nusinersen has already agreed in more than 40 countries around the world, and in 30 it already has a repayment.4 This data refers to more than 6,600 patients treated worldwide with this drug4
Nusinersen results in patients with SMA
This medicine has shown improvements in two permanent and motorized patients with SMA, the main problems that the disease gives.1 In this regard, Spain's consent is based on the results of its two basic investigations: DEVELOPMENT5 (AME from the early part) and CHERISH6 (Dave at AME), which reaches a significant clinical efficiency and favorable safety image.1
In terms of full-time SMA patients who were initially introduced (included in the ENDEAR inspection), 89 continued in the SHINE inspection,7 measured on safety and long-term nusinersen medicine.7 The results showed additional improvements in total and specific actions (such as head and seated control), together with the # 39; general motorized activity measured by CHOP INTEND.7 The safety results were consistent with those previously reported for nusinersen.7 This starting data supports the favorable profile for nusinersen risk benefits in early beginnings and patients; shows that improvements in motor milestones can be achieved despite age in medicine.7
Regarding the CHERISH study,6 Nusinersen also demonstrated clinical and statistical development clinically in motorized motoristic features with AME. Repeat delay compared to those not treated.6 The drug gradually developed and maintained, long-term motor milestones of patients.6
In addition, Biogen has a clinical program at AME as EMBRACE8 and NURTURE.9 In the NURTURE interim media analysis in a successful patient genetically tested, the cure allowed the patient to reach appropriate motor development at age and which was consistent with the development of a healthy patient.9 In this regard, 100% of the patients involved in the study could not stand without support, 88% walked with help, and 77% were absent; walk alone. In addition, all partners continued to make progress during the inspection without any evidence of sustainable reversion.10
Treating an adult patient with SMA and developing it from the cure
This misleading disease can die in its most difficult forms, especially the type of SMA 1 that appears in youth.11 According to experts, this type of patient does not normally have to reach more than two years of age due to the motive and respiratory problem caused by the disease.
However, there is also a percentage of patients with the shape of illness that is growing later, reach adult status.12 In this group of people, the disease causes a series of limitations to be particularly important when they occur; Performing basic actions such as walking and breathing, muscle impairment.11 This can reduce your hope and way of life.
By joining this medicine, adult patients will be better or better; reinforce the state by stepping up its & # 39; disease.13 In addition, the results found in clinical trials suggest that life expectancy can be increased in the long term because they are capable of SMA's motor and respiratory problems. ; cause.1
As he said Marta Valente, Biogen Medical Director of Spain, "Fostering has been a major step and change in a patient disease course with SMA. But much remains to be done. Early detection is essential, but we can not forget those patients which is an older age of life and who can benefit from this medicine as well. The current challenge is, therefore, to ensure that health professionals professionals have the most up-to-date information and can make decisions as practicable as possible, in which we work collaboratively, sharing the same goal: improving the life of patients and their families. In addition to the An effort made in AME, our company's commitment to rare diseases also implies us to work in poles such as Advanced Supranuclear Polymers or Diotatric Sclerosis, which need help fromanti-patient from around the world "14
What's In a Muscular Spinal Atrophy? 15,16,17
SMA is a rare disease that is marked by the loss of motor neurons in a & # 39; backbone and brain prevention, which means that there are hard and advanced muscles. At the end, people with the type of SMA that are most likely to lose motor loss and to make basic tricky acts are swallowing or even breathing.
The loss or damage in the SMN1 gene generates that SMA people do not Creating sufficient protein of neonary (SMN) hazard, which is essential for the maintenance of motor neurons. Two-second, same, SMN2, are capable of making the same protein, but in smaller numbers. In patients with SMA, the illness is related to the number of SMN2 gene copies and thus to the active SMN protein capacity that they can do.
People with SMA type 1 need more rigorous care, in most cases with one or two copies, Achieving their ability to sit without support or staying more than two years without respiratory support. People with SMA type 2 and type 3, with a majority of two and four copies, and # 39; make a higher amount of SMN protein and thus have smaller SMA forms but also have a very natural nature and change and their quality of life.
At Biogen, our goal is clear: we are beginners in biology. Biogen gets out, & # 39; Developing and delivering innovative worldwide medicine for patients with very poor and neurodegenerative disease. One of the world's first biotechnology companies in the world, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and the Nobel Prize won by Walter Gilbert and Phillip Sharp, and today, a package Particularly a pharmacist to complex complex treatment of sclerosis, on & # 39; The only first treated treatment for backbone illness and its & Focusing on advanced research programs in Alzheimer's and dementia, complex sclerosis and neuroimmunology, mobility disorders, neuromuscular disorders, behavior of neurocognitive, neurocognitive disorders, pain, and ophthalmology. Biogen also produces and produces. marketing biosimilar medicines of biological advanced material.
We regularly publish information that may be relevant to our tasks on our website www.biogen.com. For more information, visit www.biogen.com and continue on social networks: Twitter, LinkedIn, Facebook and YouTube.
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- M Mercuri et al. Nusinersen versus the Control of Sham in Spinal Muscular Atrophy Later on. N Engl J Med 2018; 378: 625-35
- Castro, D and everyone. Interim Report on Safety and Effectiveness of Overdue Management with Directors in Surface Inflammation (SMA): Results from the SHINE Survey. Presentation at the show at the 23rd International World Trade Association Annual Congress. 2-6 October 2018. Mendonza, Argentina. P.170
- Acsadi et al. Safety and Effectiveness of Child Protection / Children with Blood Spray (SMA): Part 1 of ISSUE 2. 2.22nd International Annual Congress of the World Cure Society. 3-7 October 2017. St Malo, France. P.380
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- Swoboda KJ and everyone. Nusinersen in childhood that begins to cure in a significant degree of backbone (SMA): effective results and interim safety from Stage 2 STANDARDS Test. Presentation at the 23rd International Annual Congress of the World Cure Society. 2-6 October 2018. Mendonza, Argentina.
- Kolb, Stephen J., et al. "Natural history of backbone backbone." Annals neurology 82.6 (2017): 883-891
- Mercuri, Eugenio, et al. "Pattern of disease progress in type 2 and 3 SMA: Outcomes for clinical trials." Neuromuscular non-rules 26.2 (2016): 126-131
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