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0 Diseases |  |
7 Trials |
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7 Trials |  |
39 News |  |
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- | Journal: Advances in Disease-Modifying Therapeutics for Chronic Neuromuscular Disorders. (Pubmed Central) - Sep 9, 2025
Amyotrophic lateral sclerosis (ALS) remains a challenge. The two most recent FDA-approved medications, namely sodium phenylbutyrate and tofersen, may slow down the disease by a few months in a selected population but do not stop the progression of the disease.
- | miglustat / Generic mfg.
Clinical, Journal: Long-Term Open-Label Study Evaluating Oral Miglustat Treatment in Patients With Neuronal Ceroid Lipofuscinosis Type 3. (Pubmed Central) - Sep 9, 2025
Miglustat showed a favorable safety profile and was associated with a slower rate of physical decline compared with historical controls. Limitations include small sample size, genetic heterogeneity, and open-label design.
- | miglustat / Generic mfg.
Journal: Is Miglustat an Effective Treatment for CLN3 (Batten) Disease? (Pubmed Central) - Sep 9, 2025
Limitations include small sample size, genetic heterogeneity, and open-label design. No abstract available
- | miglustat / Generic mfg.
Journal: Ketogenic Diet in Niemann-Pick Type C: Insights From a Case Report. (Pubmed Central) - Sep 4, 2025
In NPC patients treated with miglustat-particularly those experiencing seizures-a ketogenic diet may support neurological stabilization. A lower ratio KD may enhance long-term compliance.
- Myozyme (alglucosidase alfa) / Sanofi
Cipaglucosidase alfa and alglucosidase alfa enzymes have similar stability at neutral pH and can be stabilized with miglustat (Exhibition & Poster 2 Event Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1868;
These data confirm that rhGAAs are less stable at the near-neutral pH of blood than in acidic conditions and further support the therapeutic rationale and mechanism of action of miglustat as the only approved enzyme stabilizer used with cipa in late-onset Pompe disease. Supported by Amicus Therapeutics, Inc.
- miglustat / Generic mfg.
208-week efficacy and safety of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: muscle function and biomarkers (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1570;
P3
Conclusion Patients with LOPD, including those previously on standard-of-care alg for a median of 7.5 years, experienced sustained and durable improvements in muscle function and biomarkers following 4 years of cipa+mig treatment, supporting the long-term benefits of cipa+mig in this population. Supported by Amicus Therapeutics, Inc.
- Pombiliti (cipaglucosidase alfa-atga) / Amicus
ATB200-19: an open-label, expanded access study of the safety and effectiveness of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease in Japan (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1569;
There were no new safety concerns. Supported by Amicus Therapeutics, Inc.
- Myozyme (alglucosidase alfa) / Sanofi
Integrating clinical evidence on cipaglucosidase alfa plus miglustat and alglucosidase alfa via a multi-level network meta-regression (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1567;
Second generation ERTs cipaglucosidase alfa with miglustat (cipa+mig) and avalglucosidase alfa are now available...Using patient-level and aggregate published data from RCTs (PROPEL, COMET, LOTS) and phase I/II and open-label extension (OLE) trials (PROPEL OLE, LOTS OLE, COMET-OLE, ATB200-02, NEO-1/-EXT), a Bayesian ML-NMR was conducted, adjusting for various baseline covariates, including previous ERT duration, to obtain relative effect estimates on 6MWD (meters [m]) and ppFVC... Cipa+mig showed a statistically significant improvement versus alg in both 6MWD and FVC independent of prior ERT exposure which became more favourable when all available evidence was used in the analysis.
- miglustat / Generic mfg.
Real world study: Assessing the impact of switching from alpha-glucosidase to cipaglucosidase alfa with miglustat on disease progression in adults with Pompe disease (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1565;
Our main limitation is small sample size, which limits interpretation of coefficients, statistical power and external validity. Further research is needed to elucidate long-term effects of switching to second-generation ERT.
- Phenotypic Expression and Drug screening using NGN2-induced Neurons derived from Niemann Pick C (NPC)-iPSCs (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1549;
Lacosamide and phenelzine emerged as promising therapeutic candidates for NPC treatment. This in vitro system provides a valuable platform for understanding neuronal degeneration mechanisms and identifying new therapies.
- miglustat / Generic mfg.
Efficacy results across an observational trial, a double-blind randomized trial of arimoclomol in Niemann-Pick type C patients treated with miglustat, and an open-label extension phase (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1472;
P2/3
This in vitro system provides a valuable platform for understanding neuronal degeneration mechanisms and identifying new therapies. These data demonstrate that arimoclomol treatment as add on to routine clinical care with miglustat slowed NPC disease progression.
- miglustat / Generic mfg.
Niemann Pick Type C associated IBD: A paediatric case series from a single centre (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1471;
All 5 patients were female, with mean age at diagnosis of IBD 6.2y (range 2-11y). NPC diagnosis preceded IBD in four patients by a mean of 4.6y (range 2-6y) at a mean of 2.8y (4m-7y), all had typical NPC presentation with splenomegaly +/- hepatomegaly +/- jaundice.
- miglustat / Generic mfg.
Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to < 24 months at study enrolment (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1469;
P2/3
Arimoclomol, an orally available small molecule, is the first FDA-approved treatment for NPC when used in combination with miglustat...Overall, the evaluation of Bayley III scores showed that 1 patient gained developmental skills, 2 patients were largely stable, and 1 patient declined during the substudy. Results were inconclusive for 1 patient as Bayley III was only assessed at the baseline visit.
- miglustat / Generic mfg.
An Analysis of Biomarkers for the Evaluation of Gene Therapy for Niemann-Pick Disease Type C (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1467;
Although miglustat is an approved treatment, its effectiveness remains insufficient...PPCS and lyso-SM are considered disease biomarkers in NPC1 mice. In addition, PPCS has been suggested to be a potential biomarker for monitoring the effects of gene therapy.
- miglustat / Generic mfg.
Miglustat as a treatment for adults with Tangier Disease Neuropathy: the MUSTANG N (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1297;
In addition, PPCS has been suggested to be a potential biomarker for monitoring the effects of gene therapy. Over 2
- Myozyme (alglucosidase alfa) / Sanofi
Cipaglucosidase alfa and alglucosidase alfa enzymes have similar stability at neutral pH and can be stabilized with miglustat (Exhibition & Poster 2 Event Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1200;
These data confirm that rhGAAs are less stable at the near-neutral pH of blood than in acidic conditions and further support the therapeutic rationale and mechanism of action of miglustat as the only approved enzyme stabilizer used with cipa in late-onset Pompe disease. Supported by Amicus Therapeutics, Inc.
- miglustat / Generic mfg.
208-week efficacy and safety of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: muscle function and biomarkers (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_902;
P3
Conclusion Patients with LOPD, including those previously on standard-of-care alg for a median of 7.5 years, experienced sustained and durable improvements in muscle function and biomarkers following 4 years of cipa+mig treatment, supporting the long-term benefits of cipa+mig in this population. Supported by Amicus Therapeutics, Inc.
- Pombiliti (cipaglucosidase alfa-atga) / Amicus
ATB200-19: an open-label, expanded access study of the safety and effectiveness of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease in Japan (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_901;
There were no new safety concerns. Supported by Amicus Therapeutics, Inc.
- Myozyme (alglucosidase alfa) / Sanofi
Integrating clinical evidence on cipaglucosidase alfa plus miglustat and alglucosidase alfa via a multi-level network meta-regression (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_899;
Second generation ERTs cipaglucosidase alfa with miglustat (cipa+mig) and avalglucosidase alfa are now available...Using patient-level and aggregate published data from RCTs (PROPEL, COMET, LOTS) and phase I/II and open-label extension (OLE) trials (PROPEL OLE, LOTS OLE, COMET-OLE, ATB200-02, NEO-1/-EXT), a Bayesian ML-NMR was conducted, adjusting for various baseline covariates, including previous ERT duration, to obtain relative effect estimates on 6MWD (meters [m]) and ppFVC... Cipa+mig showed a statistically significant improvement versus alg in both 6MWD and FVC independent of prior ERT exposure which became more favourable when all available evidence was used in the analysis.
- miglustat / Generic mfg.
Real world study: Assessing the impact of switching from alpha-glucosidase to cipaglucosidase alfa with miglustat on disease progression in adults with Pompe disease (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_897;
Our main limitation is small sample size, which limits interpretation of coefficients, statistical power and external validity. Further research is needed to elucidate long-term effects of switching to second-generation ERT.
- Phenotypic Expression and Drug screening using NGN2-induced Neurons derived from Niemann Pick C (NPC)-iPSCs (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_881;
Lacosamide and phenelzine emerged as promising therapeutic candidates for NPC treatment. This in vitro system provides a valuable platform for understanding neuronal degeneration mechanisms and identifying new therapies.
- miglustat / Generic mfg.
Efficacy results across an observational trial, a double-blind randomized trial of arimoclomol in Niemann-Pick type C patients treated with miglustat, and an open-label extension phase (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_804;
P2/3
This in vitro system provides a valuable platform for understanding neuronal degeneration mechanisms and identifying new therapies. These data demonstrate that arimoclomol treatment as add on to routine clinical care with miglustat slowed NPC disease progression.
- miglustat / Generic mfg.
Niemann Pick Type C associated IBD: A paediatric case series from a single centre (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_803;
All 5 patients were female, with mean age at diagnosis of IBD 6.2y (range 2-11y). NPC diagnosis preceded IBD in four patients by a mean of 4.6y (range 2-6y) at a mean of 2.8y (4m-7y), all had typical NPC presentation with splenomegaly +/- hepatomegaly +/- jaundice.
- miglustat / Generic mfg.
Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to < 24 months at study enrolment (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_801;
P2/3
Arimoclomol, an orally available small molecule, is the first FDA-approved treatment for NPC when used in combination with miglustat...Overall, the evaluation of Bayley III scores showed that 1 patient gained developmental skills, 2 patients were largely stable, and 1 patient declined during the substudy. Results were inconclusive for 1 patient as Bayley III was only assessed at the baseline visit.
- miglustat / Generic mfg.
An Analysis of Biomarkers for the Evaluation of Gene Therapy for Niemann-Pick Disease Type C (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_799;
Although miglustat is an approved treatment, its effectiveness remains insufficient...PPCS and lyso-SM are considered disease biomarkers in NPC1 mice. In addition, PPCS has been suggested to be a potential biomarker for monitoring the effects of gene therapy.
- miglustat / Generic mfg.
Miglustat as a treatment for adults with Tangier Disease Neuropathy: the MUSTANG N (Poster 1 Annex Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_629;
In addition, PPCS has been suggested to be a potential biomarker for monitoring the effects of gene therapy. Over 2
- Miplyffa (arimoclomol) / Orphazyme, Zevra Therap
Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network (Best Poster Event Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_406;
Arimoclomol raised the level of a mature form of NPC1 protein and decreased unesterified cholesterol in the lysosomes of NPC fibroblasts. Arimoclomol activation of TFEB/TFE3 resulted in the upregulation of CLEAR gene expression including NPC1 .
- Miplyffa (arimoclomol) / Orphazyme, Zevra Therap
Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network (Best Poster Event Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_389;
Arimoclomol raised the level of a mature form of NPC1 protein and decreased unesterified cholesterol in the lysosomes of NPC fibroblasts. Arimoclomol activation of TFEB/TFE3 resulted in the upregulation of CLEAR gene expression including NPC1 .
- miglustat / Generic mfg.
Modeling Niemann-Pick Disease Type C by Directly Converted Neurons from Urine-Derived Cells (Best Poster Event Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_306;
Miglustat (N-butyldeoxynojirimycin), a glucosylceramide synthase inhibitor, is the only approved oral treatment for NPC in Japan...Some compounds significantly ameliorated the decreased survival and lysosomal dysfunction of NPC UDC-iNs, suggesting their therapeutic potential for NPC. These results suggest that UDC-iNs from disease patients can serve as a robust human cellular model for neurodegenerative disorders.
- miglustat / Generic mfg.
Modeling Niemann-Pick Disease Type C by Directly Converted Neurons from Urine-Derived Cells (Best Poster Event Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_289;
Miglustat (N-butyldeoxynojirimycin), a glucosylceramide synthase inhibitor, is the only approved oral treatment for NPC in Japan...Some compounds significantly ameliorated the decreased survival and lysosomal dysfunction of NPC UDC-iNs, suggesting their therapeutic potential for NPC. These results suggest that UDC-iNs from disease patients can serve as a robust human cellular model for neurodegenerative disorders.
- miglustat / Generic mfg.
Derivation of iPSC-Based Human Neural Models for Investigating the Pathophysiology of Sialidosis (Room 1 Main Hall) - Aug 28, 2025 - Abstract #ICIEM2025ICIEM_156;
Accumulation of sialic acid at the neuronal level was observed, underscoring its critical role in the pathogenesis of sialidosis. These pathophysiological features were compared with two iPSC-derived neuronal models from healthy controls.
- | miglustat / Generic mfg.
Biomarker, Journal: Serum neurofilament light protein as a biomarker in Niemann-Pick disease, type C1. (Pubmed Central) - Aug 18, 2025
Longitudinal analyses reveal a 26% reduction in serum NfL levels in individuals on miglustat, a therapeutic drug used off-label for the treatment of NPC1 in the United States of America...To help inform clinical trial design, our modeling predicts that a measurable reduction of serum NfL levels might be observed after 8 months of treatment with a potential drug exhibiting 10% to 20% efficacy. Our data suggest that NfL may be a useful serum biomarker for NPC1.
- | miglustat / Generic mfg.
Biomarker, Review, Journal: Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment. (Pubmed Central) - Aug 15, 2025
Therapeutic development for NPC1 has been historically limited, with miglustat approved in some regions for off-label use and 2-hydroxypropyl-?-cyclodextrin currently under clinical investigation...Ongoing clinical trials and translational research are essential to accelerate biomarker qualification and regulatory approval of disease-modifying treatments. A comprehensive, mechanistically driven approach that integrates molecular, biochemical, and clinical endpoints is key to advancing precision medicine for NPC1.
- ||| miglustat / Generic mfg.
P2/3 data, Journal: Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial. (Pubmed Central) - Jul 30, 2025
P2/3
A comprehensive, mechanistically driven approach that integrates molecular, biochemical, and clinical endpoints is key to advancing precision medicine for NPC1. The OLE of the NPC-002 trial provides evidence for a sustained reduction in disease progression for at least 5
- || Myozyme (alglucosidase alfa) / Sanofi
Clinical, Retrospective data, Journal: Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL. (Pubmed Central) - Jul 29, 2025
P3
The OLE of the NPC-002 trial provides evidence for a sustained reduction in disease progression for at least 5 ERT-experienced patients with LOPD who switched from alg to cipa+mig treatment achieved improvements or stability in most outcomes.
- | Nypta (tideglusib) / ASD Therap, miglustat / Generic mfg.
Journal: Naringenin and SMER28 target lysosomal reformation and rescue SPG11 and SPG15 Hereditary Spastic Paraplegia phenotypes. (Pubmed Central) - Jul 28, 2025
Their effects were compared with those of SMER28 and of miglustat, the latter tested in a phase II clinical trial in SPG11 patients, in both SPG15 and SPG11 patient's derived cells and in the corresponding Drosophila models...Both compounds induced lysosomal tubulation, downstream of mTOR, promoting lysosomal reformation. Our work indicates that lysosomal reformation is a good strategy for HSPs with impaired lysosomal function and identifies naringenin as new modulator of this process, offering further hand to planning phase II clinical trials in SPG11-SPG15 patients.
- || miglustat / Generic mfg.
Clinical, Journal: Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale. (Pubmed Central) - Jun 16, 2025
P2/3
Arimoclomol has been approved in the US for treatment of Niemann-Pick disease type C (NPC) in combination with miglustat...The R4DNPCCSS is a valid and reliable measure of disease progression demonstrating consistent outcomes with the prespecified 5DNPCCSS endpoint. Arimoclomol significantly slowed disease progression through 12
- miglustat / Generic mfg.
Trial completion: BBDF101-01: An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease (clinicaltrials.gov) - Jun 13, 2025
P1/2, N=6, Completed, Sponsor: Beyond Batten Disease Foundation
Arimoclomol significantly slowed disease progression through 12 Active, not recruiting --> Completed
- | Myozyme (alglucosidase alfa) / Sanofi, Nexviazyme (avalglucosidase alfa) / Sanofi, miglustat / Generic mfg.
Journal, Real-world evidence: Enzyme replacement therapies in adults with Pompe disease: from trials to real-world data. (Pubmed Central) - Jun 5, 2025
Clinical trials and early real-world data suggest that they may be superior to alglucosidase alfa, the standard of care for the past 20?years, although head-to-head comparisons between all three treatments are lacking. More data will become available over the next 5 years, leading to better guidelines for starting, stopping and switching therapies based on a more personalized assessment of outcomes.
- | doxycycline / Generic mfg., miglustat / Generic mfg.
Journal: Neurogenin 2-induced central neurons from iPSC-established patients with NPC display attenuated neurite outgrowth while accumulating cholesterol. (Pubmed Central) - May 28, 2025
Five iPSCs (3 NPC and 2 healthy individuals) carrying a doxycycline-inducible NGN2 (iPSCsTetON:NGN2) were generated, and edited cells efficiently differentiated into cortical neurons by 15?days...Miglustat, a drug approved for NPC in several countries, promoted neurite outgrowth and reduced unesterified cholesterol accumulation in LDL-treated NPC neurons...Collectively, our results indicate that neurons of NPC patients fail neurite extension due to suboptimal cholesterol transport to the membrane. This will be a valuable tool for NPC research to identify the pathological mechanisms of neuronal degeneration and to discover new therapeutics.
- | Miplyffa (arimoclomol) / Orphazyme, Zevra Therap
Journal: Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease. (Pubmed Central) - Apr 27, 2025
Arimoclomol, an orally available small molecule, is the first FDA-approved treatment for NPC when used in combination with miglustat...By also amplifying expression of CLEAR genes associated with autophagy, arimoclomol has the potential to act on different pathways and improve cell viability independent of NPC1 protein levels and functionality. In summary, the findings presented illustrate how arimoclomol improves lysosomal function and potentially autophagy flux to decrease lipid burden in NPC patient fibroblasts.
- | Journal: Assessment of repurposed compounds against coronaviruses highlights the antiviral broad-spectrum activity of host-targeting iminosugars and confirms the activity of potent directly acting antivirals. (Pubmed Central) - Apr 19, 2025
Its activity also extended to another betacoronavirus HCoV OC43, but not alphacoronavirus HCoV 229E. Our cellular screening results add to the body of knowledge on antivirals against coronaviruses and confirm the antiviral efficacy of iminosugars in cellular assays using the human lung-cell line Calu-3.
- | Myozyme (alglucosidase alfa) / Sanofi, miglustat / Generic mfg., Cabaletta (trehalose) / Seelos Therap
Journal: Miglustat: A First-In-Class Enzyme Stabilizer for Late-Onset Pompe Disease (P10-2.012). (Pubmed Central) - Apr 7, 2025
In a head-to-head study, cipa+mig had a similar safety profile to alglucosidase alfa...Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cabaletta Bio...Prof. parenti has nothing to disclose.
- | miglustat / Generic mfg.
Journal: Hypometabolism and atrophy patterns associated with Niemann-Pick type C. (Pubmed Central) - Feb 26, 2025
parenti has nothing to disclose. Our findings delineate a discernible hypometabolism pattern specific to NP-C that distinguishes it from other neurodegenerative conditions, thus suggesting that [18F]FDG PET might be a promising tool for NP-C diagnosis and to study disease progression.
- | miglustat / Generic mfg.
Journal: Niemann-Pick C-like Endolysosomal Dysfunction in DHDDS Patient Cells, a Congenital Disorder of Glycosylation, Can Be Treated with Miglustat. (Pubmed Central) - Feb 26, 2025
Treatment of DHDDS cells, with the approved NPC small molecule therapy, miglustat, improves these disease-associated phenotypes, identifying a possible therapeutic option for DHDDS patients. These data suggest that treatment options currently approved for NPC disease may be translatable to DHDDS/NUS1 patients.
- || Pombiliti (cipaglucosidase alfa-atga) / Amicus
Clinical, Journal: Effect Size Analysis of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa in ERT-experienced Adults with Late-onset Pompe Disease in PROPEL (S21.003). (Pubmed Central) - Feb 20, 2025
P3
The randomized, double-blind PROPEL study (ATB200-03; NCT03729362) compared the efficacy and safety of the investigational two-component enzyme replacement therapy (ERT) cipa+mig with alg plus placebo in adults with late-onset Pompe disease (LOPD); 77% of patients had received ERT with alg before study entry (median ERT duration 7.4 years)...Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio...Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.
- || Miplyffa (arimoclomol) / Orphazyme, Zevra Therap
Review, Journal: Arimoclomol: First Approval. (Pubmed Central) - Jan 17, 2025
Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH. Arimoclomol (MIPLYFFA
- | Pombiliti (cipaglucosidase alfa-atga) / Amicus, Myozyme (alglucosidase alfa) / Sanofi, miglustat / Generic mfg.
Journal: Challenges in multinational rare disease clinical studies during COVID-19: regulatory assessment of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease. (Pubmed Central) - Jan 12, 2025
P3
Both statistical analysis approaches led to similar results and consistent conclusions, confirming the efficacy of cipa?+?mig for adults with LOPD. NCT03729362; trial start date: December 4, 2018.Trial registration number.
- | dipyridamole / Generic mfg.
Preclinical, Journal: Dipyridamole Ameliorates Memory Impairment and Increases Hippocampal Calbindin Expression in Niemann Pick C1 Mice. (Pubmed Central) - Dec 24, 2024
Our results demonstrated that DIP selectively ameliorates the cognitive impairment in NPC1 mice. This drug could thus represent a valuable therapeutic tool to be used in combination with other treatments in NPC1.
- | miglustat / Generic mfg.
Retrospective data, Journal: Splenomegaly and progressive neurologic involvement: Think about Niemann-Pick type C disease. (Pubmed Central) - Dec 19, 2024
Niemann-Pick type C disease should be considered in the presence of visceral organ involvement and progressive neurological findings. It is essential to support the diagnosis with laboratory and genetic analysis.