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Long-Term Follow-Up before and during Riluzole Treatment in Six Patients from Two Families with Spinocerebellar Ataxia Type 7.
Suppiej, Agnese; Ceccato, Chiara; Tzekov, Radouil; Cermakova, Iveta; Parmeggiani, Francesco; Bellucci, Gianmarco; Salvetti, Marco; Zesiewicz, Theresa; Ristori, Giovanni; Romano, Silvia.
Afiliación
  • Suppiej A; Department of Medical Sciences, University of Ferrara, Ferrara, Italy. agnese.suppiej@unife.it.
  • Ceccato C; Robert Hollman Foundation, Padova, Italy. agnese.suppiej@unife.it.
  • Tzekov R; ERN-EYE Network - Center for Retinitis Pigmentosa of Veneto Region, Camposampiero Hospital, Camposampiero (Padova), Italy. agnese.suppiej@unife.it.
  • Cermakova I; Department of Medical Sciences, University of Ferrara, Ferrara, Italy.
  • Parmeggiani F; Robert Hollman Foundation, Padova, Italy.
  • Bellucci G; Department of Ophthalmology, University of South Florida, Tampa, FL, USA.
  • Salvetti M; Robert Hollman Foundation, Padova, Italy.
  • Zesiewicz T; ERN-EYE Network - Center for Retinitis Pigmentosa of Veneto Region, Camposampiero Hospital, Camposampiero (Padova), Italy.
  • Ristori G; Department of Translational Medicine for Romagna, University of Ferrara, Ferrara, Italy.
  • Romano S; Center for Experimental Neurological Therapies, Department of Neurosciences, Mental Health and Sensory Organs (NESMOS), Faculty of Medicine and Psychology, Sant'Andrea Hospital, Sapienza University of Rome, Rome, Italy.
Cerebellum ; 2024 Jul 08.
Article en En | MEDLINE | ID: mdl-38976217
ABSTRACT

BACKGROUND:

Currently no curative treatment exists for spinocerebellar ataxias (SCAs). Riluzole repurposing was proposed as a symptomatic treatment in different types of cerebellar ataxia. We report a long-term-follow up under riluzole treatment in SCA type 7.

METHODS:

Six patients received Riluzole 50 mg twice daily on a compassionate use program for a mean of 4.8 years (range 3.5-9). We measured ataxia onset and progression through the Scale for the Assessment and Rating of Ataxia (SARA), and collected extensive ophthalmological data before and after Riluzole treatment. Electrocardiogram and laboratory profile for drug safety were performed every six months.

RESULTS:

Riluzole treatment showed no effect on visual function in two patients with an advanced retinal damage. Improvements of visual function occurred in four patients followed by ophthalmologic stability up to 5 years after starting treatment. Two patients had a less steep deterioration of ataxia after treatment compared to pre-treatment, during the first 2,5 years of therapy. One showed soon after therapy an improvement of the SARA score, and then overall stability lasting 3,5 years, followed by ataxia worsening. One visually impaired patient without neurological impairment did not worse until the last visit after 3,5 years of follow-up. The remaining 2 patients showed an improvement of SARA scores soon after therapy, and an overall stability lasting respectively 5 and 3 years. No adverse event was registered during the observation period.

DISCUSSION:

This study suggests a possible beneficial action of Riluzole in SCA7 and provides a detailed description of the ophthalmologic profile of these patients.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Cerebellum Asunto de la revista: CEREBRO Año: 2024 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Cerebellum Asunto de la revista: CEREBRO Año: 2024 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Estados Unidos