baclofen (Ozobax)

[Comparison of the side effect profiles of compounds with central muscle-relaxing effect].

Kopitkó Csaba C, Pettendi Éva É

In addition to other conditions involving increased muscle spasms, centrally acting muscle relaxants (guaifenesin, tolperisone, methocarbamol, chlorzoxazone, diazepam, baclofen and tizanidin) provide relief for a significant proportion of patients suffering from low back pain. Of these, tolperisone has recently been restricted by official decision due to the high incidence of anaphylactic reactions associated with its use. The aim of our work is to analyze the frequency of side effects of this group of drugs in order to help clinicians choose the appropriate medication. According to data from the World Health Organization, tolperisone is considered a safe drug, except for the aforementioned serious allergic side effect. Mortality (including suicide) associated with the administration of these medicines is the highest among the reported side effects for diazepam, followed by tizanidine and methocarbamol. Ineffectiveness was most commonly reported for guaifenesin, followed by baclofen and tizanidine. Tolperisone was the most favorable drug in terms of both mortality and ineffectiveness. We present in detail the gastrointestinal, major neurological, and psychiatric side effects that make it difficult, and in some cases impossible, to take the drugs, and we also mention the disturbing skin and eye symptoms. Orv Hetil. 2026; 167(31): 1224-1230.

[The debate on baclofen deserves better than trials by suspicion].

Rolland Benjamin B, Karila Laurent L, Franchitto Nicolas N, Barrault Camille C et al.

Personalized Drug Repurposing Screen Identifies Patient-Specific Therapeutic Candidates for Mucopolysaccharidosis Type IIIB.

McDaniel Kathleen D KD, Ghousifam Neda N, Bowling Rodney A RA, Chen Catherine Z CZ et al.

Background: Mucopolysaccharidosis type IIIB (MPSIIIB, Sanfilippo syndrome type B) is a rare lysosomal storage disease caused by deficiency of alpha-N-acetylglucosaminidase (NAGLU) enzyme, leading to progressive accumulation of heparan sulfate and severe neurological decline. MPSIIIB's significant genetic heterogeneity presents a major barrier to developing broadly effective treatments and suggests a need for personalized therapeutic strategies. Methods: We established a personalized drug repurposing platform using high-content imaging with lysotracker dye as an indirect functional readout of lysosomal dysfunction to screen compounds that correct lysosomal defects in patient-derived fibroblasts. We screened 2807 compounds on cells from an MPSIIIB patient with a homozygous NAGLU p.Arg297Ter mutation. Hits that reduced lysosomal accumulation by at least 25% with minimal cytotoxicity were validated and subsequently tested for efficacy in fibroblasts from a second patient with a different, compound heterozygous NAGLU genotype. Results: The primary screen yielded 72 hits (2.6% hit rate), with 10 confirmed in dose-response assays. Notably, four clinically approved drugs-baclofen, dextrose, epalrestat and moxifloxacin-reduced lysosomal accumulation in the index patient's cells. However, none of these four drugs were effective in the second patient's cells, demonstrating a profound patient-specific effect. Only one non-clinical compound, 6-chlorothymol, showed a trend toward activity in both cell lines. Conclusions: Our study demonstrates a feasible framework for conducting rapid, N-of-1 drug repurposing screens for rare diseases. While we identified four promising candidates for the index patient, the lack of efficacy in a second patient cell line underscores that genetic heterogeneity may preclude a "one-size-fits-all" approach for MPSIIIB. These findings support the integration of individualized drug screening as a potential precision-medicine strategy, offering a potential path toward patient-specific therapies for rare diseases rather than traditional drug development.

Six approved drugs for alcohol use disorder: algorithms for their use according to the aims of treatment.

Caputo Fabio F, Vignoli Teo T, Lungaro Lisa L, Costanzini Anna A et al.

The pharmacological management of alcohol use disorder (AUD) currently includes six medications approved by different regulatory agencies worldwide: acamprosate (ACM), naltrexone (NTX), nalmefene (NMF), disulfiram (DF), baclofen, and sodium oxybate (SO). Their optimal use should be aligned with the main aims of treatment, namely reduction of alcohol consumption and maintenance of complete abstinence. This brief review summarizes the role of these six approved medications in AUD treatment and proposes practical treatment algorithms according to treatment goals, mechanisms of action, and clinically relevant outcomes. A focused narrative review of the literature was conducted, prioritizing meta-analyses, systematic reviews, randomized controlled trials, and relevant regulatory documents. Evidence on efficacy, safety, tolerability, and clinical applicability was interpreted pragmatically to support first-, second-, and third-line pharmacological strategies. For reducing alcohol intake, NMF or NTX are proposed as first-line pharmacotherapies, with baclofen considered as a second-line option when these agents are partially effective or ineffective. For maintaining abstinence, ACM is proposed as a first-line strategy. In selected clinical settings, including severe AUD, protracted alcohol withdrawal syndrome, high motivation for aversive treatment, or liver disease, SO, baclofen, DF, or NTX may serve as second-line options. Combined pharmacotherapy may be considered in highly adherent patients, whereas off-label drugs remain third-line options for refractory cases. Improved knowledge of the efficacy, safety, and tolerability of the six approved medications may support more appropriate, individualized, and goal-oriented pharmacological management of patients with AUD. The proposed algorithms are intended as pragmatic tools to aid clinical decision-making rather than as formal guidelines.