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- Heard, J. M., et al.
(författare)
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Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network
- 2020
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Ingår i: Orphanet Journal of Rare Diseases. - : Springer Science and Business Media LLC. - 1750-1172. ; 15:1
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Tidskriftsartikel (refereegranskat)abstract
- Background The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this field, MetabERN, the European Reference Network for hereditary metabolic diseases, performed a survey asking health care providers from 18 European countries whether these products are available on the market, reimbursed and therefore accessible for prescription, and actually delivered in their centre. Results Responses received from 52 centres (75%) concerned the design of treatment plans, the access to marketed products, and the barriers to delivery. Treatment options are always discussed with patients, who are often involved in their treatment plan. Most products (26/28) are available in most countries (15/18). Among the 15 broadly accessible products (88.5% of the centres), 9 are delivered to most patients (mean 70.1%), and the others to only few (16.5%). Among the 10 less accessible products (40.2% of the centres), 6 are delivered to many patients (66.7%), and 4 are rarely used (6.3%). Information was missing for 3 products. Delay between prescription and delivery is on average one month. Beside the lack of availability or accessibility, the most frequent reasons for not prescribing a treatment are patients' clinical status, characteristic, and personal choice. Conclusions Data collected from health care providers in the MetabERN network indicate that two-third of the orphan medicines approved by EMA for the treatment of hereditary metabolic diseases are accessible to treating patients, although often less than one-half of the patients with the relevant conditions actually received the approved product to treat their disease. Thus, in spite of the remarkable achievement of many products, patients concerned by EMA-approved orphan medicinal products have persistent unmet needs, which deserve consideration. The enormous investments made by the companies to develop products, and the high financial burden for the Member States to purchase these products emphasize the importance of a scrupulous appreciation of treatment value involving all stakeholders at early stage of development, before marketing authorization, and during follow up.
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| 2. |
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| 3. |
- Boyko, A. A., et al.
(författare)
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Intracavity difference-frequency mixing of OPO signal and idler pulses in BaGa4Se7
- 2017
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Ingår i: Optics InfoBase Conference Papers. - : Optical Society of America. - 9781943580279
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Konferensbidrag (refereegranskat)abstract
- An overall quantum conversion efficiency of 7.8% is achieved by intracavity mixing the signal and idler of a 1.064-μm pumped Rb:PPKTP OPO in BaGa4Se7, generating >0.7 mJ pulse energy at ~7 μm and 100 Hz.
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| 4. |
- Petrov, V., et al.
(författare)
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Frequency down-conversion of 1 μm laser radiation to the mid-IR using non-oxide nonlinear crystals in a cascaded intracavity configuration
- 2017
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Ingår i: Proceedings of SPIE - The International Society for Optical Engineering. - : SPIE - International Society for Optical Engineering. - 9781510606173
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Konferensbidrag (refereegranskat)abstract
- A singly-resonant OPO (SRO) based on AgGaSe2 (AGSe) intracavity pumped at ∼1.85 μm by the signal pulses of a Rb:PPKTP doubly-resonant OPO (DRO) provided extremely broad tuning (5.8 to ∼18 μm) for the non-resonated idler. In a similar set-up with the same nonlinear crystals, we studied intracavity difference-frequency generation (DFG). Both AGSe and the new monoclinic crystal BaGa4Se7 (BGSe) generated single pulse energies of ∼0.7 mJ near 7 μm at an overall conversion efficiency from the 1.064 μm pump of 1.2%. The main advantage of BGSe is its damage resistivity up to the maximum pump levels applied at 100 Hz.
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| 6. |
- Boyko, A. A., et al.
(författare)
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Intracavity-pumped, cascaded Mid-IR optical parametric oscillator based on AgGaSe2
- 2015
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Ingår i: Advanced Solid State Lasers, ASSL 2015. - : Optical Society of America. - 9781943580026
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Konferensbidrag (refereegranskat)abstract
- We report on an AgGaSe2 optical parametric oscillator (OPO), intracavity pumped by the 1.85-μm signal pulses from a 1.064-μm pumped Rb:PPKTP OPO. It operates at 100 Hz with idler tunability from 5.8 to 8.3 μm.
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