News
03. März 2026 – Proposed pharmacovigilance algorithm DMD
Proposed pharmacovigilance algorithm for children and adolescents with Duchenne muscular dystrophy (DMD): shown are the proposed time intervals and examinations
Stand: Dez 2025
Table1: Standard of Care (SoC) and/or therapy with vamorolon Abbreviations: FC= first contact, W=week, M=month
| FC | Once per 6 months | Once per year | Once per 2 years | |
| Mol.-Genetic confirmation1 | ● | |||
| Physical examination | ● | ● | ||
| Anthropometry, RR, HR | ● | ● | ||
| Education2 | ● | (●) | (●) | |
| Genetic counceling3 | ● | |||
| Check vaccination4 | ● | (●) | (●) | |
| Laboratory investigations5 | ● | ● | ||
| Echocardiography/ECG/cardiac prophylaxis6 | ● | (●) | ● | |
| Cardiac MRI7 | (●) | |||
| Lung function8 | ● | (●) | ● | |
| Lateral spine X ray/DXA/pQCT9 | (●) | ● | ||
| Motor function status10 | ● | (●) | ● | |
| PROMS (DMD-QoL, EQ5d/5L) | ● | ● | ||
| Cognition/Behavioral screening 11 | ● | (●) | ||
| Cognition/Speech testing 12 | ● | (●) | ||
| Social worker13 | (●) | (●) | ||
| Information on registry | ● | |||
| Registry documentation | ● | ● |
2 Information on natural course of DMD, treatment options and side effects
3 Genetic counseling and carrier screening of mother, if positive -> recommend cardiologic follow-up for the mother
4 Check for completeness of vaccination status according to STIKO recommendations, especially with life vaccines prior to steroid treatment; additionally, 23-valent pneumococcus vaccination and yearly seasonal influenca vaccination
5 Once/year: full blood count, alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma gluraryl transferase (GGT), bilirubin, cystatin C, triglycerides, cholesterol, hemoglobin A1C (HbA1c), 25-OH-cholecalerol, calcium/creatinine ration in urin; facultative: calcium, phosphate, sodium, potassium, creatinkinase, parathyroid hormone, thyroid-stimulating hormone (TSH), thyroxine (fT4), bone specific alkaline phosphatase, osteocalcin, ß-crosslinks (ß-CTX), procollagen type 1 N propeptide (P1NP), tartrate-resistent acid phosphatase 5b (TRAP 5b), N-terminal pro-B-type natriuretic peptide (NTProBNP), insulin-like growth factor 1 (IGF-1), insuline-like growth factor binding protein 3 (IGF-BP3), pyridinolin (PYD) and deoxypyridinoline (DPD) in urine; from 13 years onwards: luteinizing hormone (LH), follicle-stimulating hormone (FSH), testosterone
6 In ambulatory patients once/year; increase frequency based on cardiologist’s recommendation + prior to planned anaesthesia; 24-h Holter ECG in case of left ventricular dysfunction/based on cardiologist’s recommendation
7 Consider Cardio MRI at 7 years if possible without anaesthesia; interval at cardiologist’s recommendation
8 in patients >8 years: 1x/year; after loss of ambulation: 2x/year; sleep study when FVC ≤50% or clinical signs of nocturnal hypoventilation
9 from 10 years on every 18-24 months: lateral spine X-ray (Genant score), alternatively dual energy X-ray absorptiometry (DXA scan) or peripheral quantitative computed tomography (pQCT); under bisphosphonate therapy: every 12 months
10 Minimum for patients >4 years: North Star Ambulatory Assessment (NSAA) including timed function tests in ambulatory patients (TTR, 4SC, 10MWR), Performance of Upper Limb (PUL) or Egon scales in non-ambulatory patients
11 screening: CBCL and DuMAND in children > 18months; in case of abnormalities annually, otherwise at 6 and 12 years
12 in all children with DMD at presentation and at the age of 6 years; Bayley III-scales (1-42 months),
WPPSI-IV (2,6-3,11 Ys; 4-7,7 Ys), from Wechsler Intelligence Scales, WISC-V (6-16,11 Ys), from Wechsler Intelligence Scales; for non-native-speakers Wechsler Intelligence Scales (language free version
13 Timing flexible and depending on individual need: advice on grade of disability, schooling, travel costs, etc.
Table 2 additional algorithm for treatment with givinostat
Abbreviations: FC= first contact, W=week, M=month
| FC | W2 | W4 | W6 | W8 | W12 | M6 | M9 | M12 | Once per 6 months | Once per year | |
| Full blood count | ● | ● | ● | ● | ● | ● | ● | ● | ● | every 3 months | |
| Triglycerides, bilirubin, GGT, GOT, GPT | ● | ● | ● | ● | ● | ● | |||||
| Thromboplastin time (TPZ) from Plasma/Quick, Partial Thromboplastin Time (PTT), vWF, VWF Antigen, fT3, fT4, TSH, Fak.XIII, Lipase, Fibrinogen | ● | ● | ● (only coagulation) | ● (only coagulation) | ● (only coagulation) | (●) (only coagulation) | |||||
| ECG | ● | ● | ● | ● | ● |
21. Januar 2026 – Nationales Gen- und Zelltherapieregister Deutschland (nGCT-R) gegründet
GNP als Gründungsmitglied – Nationales Gen- und Zelltherapieregister Deutschland (nGCT-R) gegründet
Federführende Fachgesellschaften haben den Verein „Nationales Gen- und Zelltherapieregister Deutschland e.V. (nGCT-R)“ gegründet.
Ziel des nGCT-R ist die Verstetigung einer zentralen, digitalen Infrastruktur, die die Anwendung innovativer Gen- und Zelltherapien unabhängig erfasst. Das Register liefert eine verlässliche Datenbasis, um Wirksamkeit und Sicherheit dieser Therapien indikationsübergreifend zu bewerten und damit Forschung, Versorgung und Patient*innensicherheit zu stärken. Perspektivisch soll das Register auf weitere Fachgebiete mit Zulassungen im Bereich der Gen- und Zelltherapie ausgeweitet und Politik, Krankenkassen, Behörden sowie die Industrie strukturiert eingebunden werden.
www.ngctr.de
15. Januar 2026 – GNP-Jahrestagung in Linz
Wir freuen uns, Sie in Linz auf unserer Jahrestagung 2026 begrüssen zu dürfen! 07.-09. Mai 2026, 06. Mai 2026 FoBi-Akademie
Alle Infos zu Anmeldung, Abstract-Einreichung und Programm finden Sie auf unserer Kongress-Website
gnp-kongress.de
