natgillin/translations-raw huggingface dataset:
- Udvalget var desuden af den opfattelse, at der er blevet rapporteret et betydeligt antal dødsfald, hvor dextropropoxyfen fandtes på toksisk niveau, hvilket bekræfter, at risikoen for en utilsigtet dødelig overdosering, der kan sammenkædes med lægemidler indeholdende dextropropoxyfen samt deres snævre terapeutiske indeks, er et stort problem. 
- The Committee also considered that a significant number of deaths have been reported in which dextropropoxyphene is present at toxic levels confirming that the risk of accidental fatal overdose associated with dextropropoxyphene containing medicinal products and their narrow therapeutic index is of major concern;
Selv om spontane rapporter antydede, at sikkerhedsrisikoen i forbindelse med overdosering ikke var signifikant, bekræftede andre mere udførlige data, især fra retsmedicinske centre og nationale dødsstatistikker, at risikoen for en utilsigtet dødelig overdosering under normale anvendelsesbetingelser, der kan sammenkædes med lægemidler indeholdende dextropropoxyfen, er et stort problem, hovedsageligt på grund af deres snævre terapeutiske indeks og høje dødsrate. 
Although spontaneous reporting suggested that the safety signal concerning the overdose was not significant, other more complete data, particularly from forensic centres and national mortality statistics confirmed that the risk of accidental fatal overdose under normal conditions of use associated with dextropropoxyphene containing products is of major concern, mainly due to their narrow therapeutic index and high case fatality.
CHMP konkluderede derfor, at benefit/risk-forholdet for lægemidler indeholdende dextropropoxyfen er negativt, og at dets udtalelse af 25. juni 2009 ikke skulle revideres for orale/rektale lægemidler indeholdende dextropropoxyfen, og anbefalede en tilbagekaldelse af markedsføringstilladelserne, som skal gælde i en periode på 15 måneder fra Kommissionens beslutning for at gøre det muligt for patienter at skifte til sikrere alternativer, navnlig i betragtning af den udbredte kliniske anvendelse af lægemidler indeholdende dextropropoxyfen og den store patienteksponering i visse medlemsstater. 
Therefore, the CHMP concluded by majority that the benefit-risk balance of dextropropoxyphene containing medicinal products is negative and that its Opinion of 25 June 2009 should not be revised for oral/rectal dextropropoxyphene containing medicinal products and recommended the withdrawal of the Marketing Authorisations to be effective within the next 15 months of the Commission Decision in order to allow switching patients to safer alternatives, considering the extensive clinical use of dextropropoxyphene containing medicinal products and the wide patient exposure in some Member States.
Efter en gennemgang af den detaljerede skriftlige begrundelse for revurdering og den mundtlige redegørelse fra gruppen af indehavere af markedsføringstilladelse fandt CHMP, at udformningen af den foreslåede kliniske undersøgelse til påvisning af, at kombinationen af dextropropoxyfen og paracetamol havde bedre virkning end paracetamol alene, var fejlbehæftet, og at selv en veldesignet undersøgelse ikke ville ændre benefit/risk-forholdet for lægemidler indeholdende dextropropoxyfen i lyset af det snævre terapeutiske indeks. 
Having considered the detailed grounds for re-examination provided by the group of MAHs in writing and in an oral explanation, the CHMP considered that the design of the proposed clinical study to demonstrate the superior efficacy for combination of dextropropoxyphene and paracetamol versus paracetamol alone was flawed, and even a well-designed study would not change the benefit-risk balance of the dextropropoxyphene medicinal products in view of the narrow therapeutic index.
Nogle patienter oplever, at disse lægemidler er effektive til smertebehandling, men resultater fra kliniske undersøgelser giver ikke dokumentation for højere effekt af dextropropoxyfen alene eller i kombination med paracetamol sammenlignet med normale terapeutiske doser af simple smertestillende lægemidler. 
While some patients find these products helpful in managing pain, results from clinical trials do not provide evidence for the superior efficacy of dextropropoxyphene alone or in combination with paracetamol, when compared with normal therapeutic doses of simple analgesics.
Efter at have gennemgået alle tilgængelige data vurderede CHMP, at de forskellige tal, der fremgår af datakilderne (spontane rapporter, retsmedicinske centre, giftinformationscentraler, nationale dødsstatistikker), generelt viste et betydeligt antal af dødsfald, hvor dextropropoxyfen fandtes på toksisk niveau. 
After reviewing all the available data, the CHMP considered that the different figures provided by the data sources (spontaneous reports, forensic and poison centres, national mortality statistics) showed overall a significant number of deaths in which dextropropoxyphene is present at toxic levels.
Medlemsstat Indehaver af markedsføringstilladelse 
Member State Marketing Authorisation Holder Product Name
De forskellige tal, der fremgår af datakilderne (spontane rapporter, retsmedicinske centre, giftinformationscentraler, nationale dødsstatistikker), viste generelt et betydeligt antal dødsfald, hvor dextropropoxyfen fandtes på toksisk niveau. 
The different figures provided by the available data sources (spontaneous reports, forensic and poison centres, national mortality statistics) showed overall a significant number of deaths in which dextropropoxyphene is present at toxic levels.
- Udvalget vurderede, at lægemidler indeholdende dextropropoxyfen kun havde begrænset effekt ved symptomatisk behandling af smerter. 
- The Committee considered that dextropropoxyphene containing medicinal products showed only limited efficacy in the symptomatic treatment of pain;
De tilgængelige data viste kun en begrænset effekt af lægemidler indeholdende dextropropoxyfen til symptomatisk behandling af smerter. 
Available data showed only limited efficacy of the dextropropoxyphene medicinal products in the symptomatic treatment of pain.
Kapsel med modificeret udløsning 
Modified release capsule Oral use
CHMP mente desuden, at de data, der er indsamlet fra de nationale giftinformationscentraler, også har begrænsninger i denne situation, da dextropropoxyfen kan være dødbringende efter ekstremt kort tid (under en time). 
The CHMP also considered that data collected from national poison centres have limitations in this situation as dextropropoxyphene can cause death extremely rapidly (in under an hour); if a patient dies before reaching medical attention, the poisons centre is unlikely to be contacted.
Forslag om at begrænse hver enkelt recept til højst 15 dage eller en måned, før den, der har udskrevet recepten, skal revidere den, vil sandsynligvis ikke få nogen betydning for risikominimeringen. 
Similarly, proposals to limit supplies for each prescription to at most 15 days, or one month, before review is needed by the prescriber, are unlikely to be of any significant benefit in risk minimisation: the patient will still have access to significant large quantity in excess of the lethal dose.
Det er ikke muligt at anvende rutinemæssigt indsamlede (spontane) data til at vurdere effektiviteten af foranstaltninger til risikominimering på grund af den betydelige underrapportering af endog alvorlige bivirkninger, herunder dødsfald. 
However no consideration was given to the need for national mortality data, and in particular forensic pathology data, to ensure that any risk minimisation measures are working: it is not possible to use routinely-collected (spontaneous) data to assess the effectiveness of the risk minimisation measures, because of the significant under-reporting of even serious adverse events, including death.
Der foreligger dog ikke tilstrækkelig dokumentation fra kliniske forsøg for, at effekten af kombinationen af dextropropoxyfen og paracetamol skulle være højere end ved normale terapeutiske doser af paracetamol. 
However, there is no clear evidence from clinical trials of superiority of efficacy of the combination of dextropropoxyphene and paracetamol compared with normal therapeutic doses of paracetamol alone, the trials which have suggested superiority to paracetamol alone have used sub-therapeutic doses of paracetamol.
Til akutte smerter var den faste kombination af dextropropoxyfen og paracetamol tilsyneladende et effektivt smertestillende lægemiddel. 
In acute pain, the fixed combination of dextropropoxyphene and paracetamol appeared to be an effective analgesic; this is to be expected, as paracetamol alone is an effective analgesic.
Dextropropoxyphene Paracetamol Theraplix 
Dextropropoxyphene Paracetamol almus
Fortegnelse over lægemidlernes navne, lægemiddelformer, styrker, indgivelsesveje, ansøgere, indehavere af markedsføringstilladelser i medlemsstaterne 
List of the names, pharmaceutical forms, strengths of the medicinal products, routes of administration, marketing authorisation holders in the Member States
Storbritannien Manx Healthcare Limited Taylor Group House 
United Kingdom Manx Healthcare Limited Taylor Group House
Storbritannien Wockhardt UK Limited Ash Road North 
United Kingdom Wockhardt UK Limited Ash Road North
Storbritannien Bristol Laboratories Limited Unit 3, Canalside 
United Kingdom Bristol Laboratories Limited Unit 3, Canalside
Storbritannien Generics (UK) Limited, T/A Mylan, Albany Gate, Potters Bar, 
United Kingdom Generics (UK) Limited, T/A Mylan, Albany Gate, Potters Bar,
Storbritannien Athlone Pharmaceuticals Limited, Ballymurray, Roscommon, Co. 
United Kingdom Athlone Pharmaceuticals Limited, Ballymurray, Roscommon, Co.
Grækenland JOHNSON & JOHNSON ΗΕΛΛΑΣ CONSUMER SA 
JOHNSON & JOHNSON ΗΕΛΛΑΣ CONSUMER SA
Storbritannien Focus Pharmaceuticals Limited Unit 5, Faraday Court 
United Kingdom Focus Pharmaceuticals Limited Unit 5, Faraday Court
mg/100 ml sirup 
mg/100 ml syrup
Storbritannien Co-Pharma Limited Unit 4, Metro Centre Tolpits Lane, Watford Hertfordshire 
United Kingdom Co-Pharma Limited Unit 4, Metro Centre Tolpits Lane, Watford Hertfordshire
Storbritannien McNeil Products Limited, Johnson & Johnson Ltd, Foundation Park, Roxborough Way, Maidenhead, Berkshire SL6 3UG, 
United Kingdom McNeil Products Limited, Johnson & Johnson Ltd, Foundation Park, Roxborough Way, Maidenhead, Berkshire SL6 3UG,
Grækenland PIERRE FABRE MEDICINES SA Messogeion 350, Agia Paraskevi 
PIERRE FABRE MEDICINES SA Messogeion 350, Agia Paraskevi
5 mg oral suspension Oral anvendelse 
5 mg oral suspension oral use
Storbritannien Medreich plc, Warwick House, Plane Tree Crescent, Feltham TW13 7HF United Kingdom domperidone maleate 
United Kingdom Medreich plc, Warwick House, Plane Tree Crescent, Feltham TW13 7HF United Kingdom domperidone maleate
UAB „Johnson & Johnson“ Geležinio Vilko g. 18A LT-08104 Vilnius Lithuania domperidone Motilium 10 mg filmovertrukket tablets 
UAB „Johnson & Johnson“ Geležinio Vilko g. 18A LT-08104 Vilnius Lithuania domperidone Motilium 10 mg filmcoated tablets
Storbritannien Co-operative Pharmacy National Distribution Centre Limited, Enterprose Way, Meir Park, Stokeon-Trent, Staffordshire ST3 7UN United Kingdom domperidone maleate 
United Kingdom Co-operative Pharmacy National Distribution Centre Limited, Enterprose Way, Meir Park, Stokeon-Trent, Staffordshire ST3 7UN United Kingdom domperidone maleate
Storbritannien Milpharm Limited, Ares Block, Odyssey Business Park, West End Road, Ruislip, HA4 6QD, United Kingdom domperidone maleate 
United Kingdom Milpharm Limited, Ares Block, Odyssey Business Park, West End Road, Ruislip, HA4 6QD, United Kingdom domperidone maleate
Rumænien S.C. TERAPIA S.A. Str. Fabricii nr. 124, Cluj Napoca, Romania 
Str. Fabricii nr. 124, Cluj Napoca, Romania
1 mg/ml oral suspension Oral anvendelse 
1 mg/ml oral suspension oral use
Belgium domperidone Motilium pediatrie 1 mg/ml oral suspension Oral anvendelse 
Belgium domperidone Motilium pediatrie 1 mg/ml oral suspension oral use
Maroussi 15125 
Epidarvou 4 & Aigialeias Maroussi 15125
Domperidone Motilium Tropfen 10 mg oral drabber, suspension 
Domperidone Motilium Tropfen 10 mg oral drops, suspension
Storbritannien Winthrop Pharmaceuticals UK Limited, One Onslow Street, Guildford, Surrey GU1 4YS, United Kingdom domperidone Domperidone 1mg/ml Oral 
United Kingdom Winthrop Pharmaceuticals UK Limited, One Onslow Street, Guildford, Surrey GU1 4YS, United Kingdom domperidone Domperidone 1mg/ml Oral
Storbritannien Winthrop Pharmaceuticals UK Limited, One Onslow Street, Guildford, Surrey GU1 4YS United Kingdom domperidone maleate 
United Kingdom Winthrop Pharmaceuticals UK Limited, One Onslow Street, Guildford, Surrey GU1 4YS United Kingdom domperidone maleate
10 mg opløselig tablet 
10 mg dispersible tablet
10 mg oroopløselig tablet Oral anvendelse 
10 mg orodispersible tablet oral use
Portugal domperidone Domperidona Mylan 10 mg filmovertrukket tablet 
Portugal domperidone Domperidona Mylan 10 mg film-coated tablet
20 mg brusegranulat 
20 mg effervescent granules
5 mg brusegranulat 
5 mg effervescent granules
Motilium 10mg Filmovertrukket Tablets 
Motilium 10mg Filmcoated Tablets
Luxembourg Johnson & Johnson Consumer N.V. 
Luxembourg Johnson & Johnson Consumer N.V. Antwerpseweg 15-17
10 mg brusegranulat 
10 mg effervescent granules
Belgium domperidone Motilium-60 supp 60 mg stikpille 
Belgium domperidone Motilium-60 supp 60 mg suppository rectal use
Belgium domperidone Motilium-30 supp 30 mg stikpille 
Belgium domperidone Motilium-30 supp 30 mg suppository rectal use
Domperidone mylan 10 mg filmovertrukket tablet 
Domperidone mylan 10 mg film-coated tablet
DOMPERIDONE MOTILIUM 10 mg 10 mg filmovertrukket tablet 
DOMPERIDONE MOTILIUM 10 mg 10 mg film-coated tablet
Belgium domperidone Motilium Instant-10 10 mg smeltetablet 
Belgium domperidone Motilium Instant-10 10 mg orodispersible tablet oral use
France domperidone DOMPERIDONE SUBSTIPHARM 20 mg, comprimé pelliculé sécable 
75116 Paris domperidone DOMPERIDONE SUBSTIPHARM 20 mg, comprimé pelliculé sécable
30 mg suppositorie 
30 mg suppository rectal use
France domperidone MOTILIUM 10 mg, granulés effervescents en sachet-dose 
92787 Issy-les-Moulineaux Cedex domperidone MOTILIUM 10 mg, granulés effervescents en sachet-dose
France domperidone DOMPERIDONE SGPHARM 20 mg, comprimé pelliculé sécable. 
75116 Paris domperidone DOMPERIDONE SGPHARM 20 mg, comprimé pelliculé sécable.
Belgium domperidone Domperidone EG-10 10 mg smeltetablet 
Belgium domperidone Domperidone EG-10 10 mg orodispersible tablet oral use
France domperidone DOMPERIDONE GENODEX 20 mg, comprimé pelliculé sécable. 
75116 Paris domperidone DOMPERIDONE GENODEX 20 mg, comprimé pelliculé sécable.
Rumænien Pierre Fabre Médicament 45 place Abel Gance 
Belgium Pierre Fabre Médicament 45 place Abel Gance
B-2340, Beerse Belgium domperidone MOTILIUM STIKPILLE 30MG 
B-2340, Beerse Belgium domperidone MOTILIUM SUPPOSITORY 30MG
Domperidone Motilium Rx 10 mg Filmovertrukket 
Domperidone Motilium Rx 10 mg Film-coated Tablets.
Kela Pharma nv Industriepark 68 9100 Sint-Niklaas Belgium 
Belgium Kela Pharma nv Industriepark 68
Domperidon teva 10 mg filmovertrukket tablet 
Domperidon teva 10 mg film-coated tablet
Pierre Fabre Médicament 45 place Abel Gance 92100 Boulogne France domperidone OROPERIDYS OROOPLØSELIG TABLETS 10MG 
Pierre Fabre Médicament 45 place Abel Gance 92100 Boulogne France domperidone OROPERIDYS ORODISPERSIBLE TABLETS 10MG
Pierre Fabre Médicament 45 place Abel Gance 92100 Boulogne France domperidone Oroperidys 10 mg 10 mg oroopløselig tablet Oral anvendelse 
Pierre Fabre Médicament 45 place Abel Gance 92100 Boulogne France domperidone Oroperidys 10 mg 10 mg orodispersible tablet oral use
Domperidone Motilium 10mg Filmovertrukk et Tablets 
Domperidone Motilium 10mg FilmCoated Tablets
Pierre Fabre Médicament 45 place Abel Gance 92100 Boulogne France domperidone Oroperidys 10 mg smeltetablets 
Pierre Fabre Médicament 45 place Abel Gance 92100 Boulogne France domperidone Oroperidys 10 mg orodispersible tablets
Motilium Instants 10 mg smeltetablet 
Motilium Instants 10 mg orodispersible tablet oral use
Dubrova 308 52220 Labin Kroatien 
Dubrova 308 52220 Labin Croatia
Dental anvendelse Perineural anvendelse Submukosal anvendelse Infiltration 
Dental use Perineural use Submucosal use Infiltration
Perineural anvendelse Infiltration 
Perineural use Infiltration
Infiltration Perineural anvendelse 
Infiltration Perineural use
3% uden vasokonstrikt or 
3% without vasoconstrictor
Laboratoires Septodont 58, rue du Pont de Créteil 94100 Saint-Maur-desFossés 
Laboratoires Septodont 58, rue du Pont de Créteil 94100 Saint-Maur-des-Fossés France
Perineural anvendelse Submukosal anvendelse 
Perineural use Submucosal use
MG/ML, SOLUTION INJECTABLE À USAGE DENTAIRE 
SCANDONEST 30 MG/ML, SOLUTION INJECTABLE À USAGE DENTAIRE
Hvad er Kyndrisa? 
What is Kyndrisa?
Tilbagetrækning af ansøgningen om markedsføringstilladelse for Kyndrisa (drisapersen) 
Withdrawal of the marketing authorisation application for Kyndrisa (drisapersen)
Kyndrisa er et lægemiddel, der indeholder det aktive stof drisapersen. 
Kyndrisa is a medicine that contains the active substance drisapersen.
Hvis du deltager i en klinisk undersøgelse med Kyndrisa og har behov for mere information om din behandling, kan du kontakte den læge, der giver dig behandlingen. 
If you are in a clinical trial and need more information about your treatment, contact the doctor who is giving it to you.
Virksomheden har oplyst, at den vil stoppe udviklingen af dette lægemiddel, men at den planlægger at samarbejde med læger, patienter og lokale lægemiddelmyndigheder om at give de aktuelt behandlede patienter mulighed for at få adgang til de resterende forsyninger af Kyndrisa. 
The company stated that it intended to stop the development of the medicine, but that it plans to work with doctors, patients and local regulatory authorities to allow currently treated patients to access the remaining supply of Kyndrisa.
Udvalget vurderede, at data fra de kliniske undersøgelser ikke i tilstrækkelig grad havde påvist en gavnlig virkning ved Kyndrisa: Hovedundersøgelsen påviste ingen virkning hos patienter med lidelsen, mens to andre undersøgelser ikke på konsekvent vis påviste en gavnlig virkning. 
The Committee considered that data from the clinical studies did not sufficiently demonstrate a beneficial effect of Kyndrisa: the main study did not show any benefit in patients with the condition, while two other studies failed to consistently show a beneficial effect.
Lægemidlet virker i kraft af "exon 51 skipping"-teknologien. 
This medicine works by ‘exon 51 skipping
Hvad forventedes Kyndrisa anvendt til? 
What was Kyndrisa expected to be used for?
Hvordan forventes Kyndrisa at virke? 
How is Kyndrisa expected to work?
Kyndrisa blev undersøgt i tre undersøgelser med i alt 290 patienter med Duchennes muskeldystrofi. 
Kyndrisa was investigated in three studies in a total of 290 patients with Duchenne muscular dystrophy.
Desuden var lægemidlets sikkerhedsprofil ikke tilfredsstillende, især på grund af risikoen for vedvarende reaktioner på injektionsstedet (f.eks. hævelse, betændelse og sårdannelse), der kunne have en negativ indvirkning på livskvaliteten, og risikoen for trombocytopeni (for lavt antal blodplader), der kunne give patienterne en betydelig risiko for alvorlige blødningskomplikationer. 
Further, the safety profile of the medicine was not considered satisfactory, particularly because of the risk of persisting reactions at the injection site (such as swelling, inflammation and ulceration) which could have a negative impact on quality of life, and the risk of thrombocytopenia (low blood platelet counts) which could put patients at risk of serious bleeding complications.
Derfor var CHMP på tidspunktet for tilbagetrækningen af den opfattelse, at fordelene ved Kyndrisa ikke opvejede risiciene. 
Therefore, at the time of the withdrawal, the CHMP was of the opinion that the benefits of Kyndrisa did not outweigh its risks.
Efter at CHMP havde vurderet virksomhedens besvarelse af den sidste række spørgsmål, var der fortsat visse vigtige uafklarede punkter. 
After the CHMP had assessed the company's responses to the last round of questions, there were still some important unresolved issues.
I sit brev til agenturet om tilbagetrækningen af ansøgningen anførte virksomheden, at den ikke ville være i stand til at besvare CHMP's betænkeligheder vedrørende resultaterne af de kliniske undersøgelser inden for den forventede tidsramme. 
In its letter notifying the Agency of the withdrawal of application, the company stated that it would not be able to address the CHMP concerns regarding the results of the clinical studies within the expected timeframe.
Den 31. maj 2016 meddelte BioMarin International Limited officielt Udvalget for Lægemidler til Mennesker (CHMP), at virksomheden ønsker at trække sin ansøgning om markedsføringstilladelse for Kyndrisa til behandling af Duchennes muskeldystrofi tilbage. 
On 31 May 2016, BioMarin International Limited officially notified the Committee for Medicinal Products for Human Use (CHMP) that it wishes to withdraw its application for a marketing authorisation for Kyndrisa, for the treatment of Duchenne muscular dystrophy.
Duchennes muskeldystrofi er en alvorlig genetisk sygdom, der primært rammer drenge, og som normalt diagnosticeres før seksårs alderen. 
Duchenne muscular dystrophy is a serious genetic disease, which mainly affects boys and is usually diagnosed before the age of 6.
Kyndrisa skulle anvendes til behandling af Duchennes muskeldystrofi hos patienter i alderen fem år og derover, som er i stand til at gå, og hvis sygdom skyldes visse genetiske forandringer (defekter), der kan behandles med "exon 51 skipping"-teknologi. 
Kyndrisa was expected to be used for the treatment of Duchenne muscular dystrophy in patients aged 5 years and older who are able to walk and whose disease is due to certain genetic mutations (defects) that can be treated with ‘exon 51 skipping technology'.
I undersøgelserne blev virkningen af Kyndrisa sammenlignet med placebo (en uvirksom behandling), og det primære mål for virkningen var ændringen i den afstand, patienten kunne gå i løbet af seks minutter efter 24 eller 48 ugers behandling. 
The studies compared the effects of Kyndrisa with placebo (a dummy treatment) and the main measure of effectiveness was the change in the distance the patient could walk in six minutes after 24 or 48 weeks of treatment.
Denne teknologi sætter den proteindannende mekanisme i cellerne i stand til at udelade visse dele af dystrofingenet og muliggør dannelsen af et forkortet, men delvist fungerende dystrofinprotein. 
This technology enables the protein-making apparatus in cells to skip some areas of the dystrophin gene and allow the production of a shortened but partially working dystrophin protein.
På baggrund af gennemgangen af dataene og virksomhedens besvarelse af CHMP's liste med spørgsmål på tidspunktet for tilbagetrækningen af ansøgningen havde CHMP visse betænkeligheder og var af den foreløbige opfattelse, at Kyndrisa ikke ville kunne være blevet godkendt til behandling af Duchennes muskeldystrofi. 
Based on the review of the data and the company's response to the CHMP lists of questions, at the time of the withdrawal, the CHMP had some concerns and was of the provisional opinion that Kyndrisa could not have been approved for the treatment of Duchenne muscular dystrophy.
Det var hensigten, at det skulle markedsføres som injektionsvæske, opløsning, til indsprøjtning under huden. 
It was to be available as a solution for injection under the skin.
Duchennes muskeldystrofi skyldes mutationer i genet for proteinet dystrofin, hvilket medfører dannelse af ikke-funktionel dystrofin. 
Duchenne muscular dystrophy is caused by mutations in the gene for the protein dystrophin, which lead to the production of a non-functional dystrophin.
Sygdommen forårsager fremadskridende svækkelse og tab af muskelfunktion, og patienterne dør typisk i teenagealderen eller i starten af deres voksenliv. 
It causes progressive weakness and loss of muscle function, and usually leads to death in adolescence or early adulthood.