| The potential of utrophin modulators for the treatment of Duchenne muscular dystrophy |
10 |
| Leishmaniasis: treatment, drug resistance and emerging therapies |
7 |
| A developing portrait of hereditary periodic fevers in childhood |
5 |
| Migalastat for the treatment of Fabry disease |
4 |
| Intrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects |
3 |
| Advances in understanding disease mechanisms and potential treatments for Crigler-Najjar syndrome |
3 |
| Paget's disease of bone: an update on epidemiology, pathogenesis and pharmacotherapy |
3 |
| Advances in new drug therapies for the management of sickle cell disease |
3 |
| Gene therapy for the treatment of X-linked retinitis pigmentosa |
3 |
| Advances in the treatment of neuronal ceroid lipofuscinosis |
2 |
| Naegleria fowleri: diagnosis, treatment options and pathogenesis |
2 |
| Emerging therapeutics for the treatment of Friedreich's ataxia |
2 |
| The potential role of gut microbiota and its modulators in the management of propionic and methylmalonic acidemia |
2 |
| Evaluating the impact of peer support and connection on the quality of life of patients with familial chylomicronemia syndrome |
2 |
| Hereditary multiple exostoses: are there new plausible treatment strategies? |
2 |
| Treatment strategies for neuroendocrine liver metastases: an update |
2 |
| Repurposing drugs for the treatment of galactosemia |
1 |
| Advances in understanding and treatment of scleromyxedema |
1 |
| The potential of emerging new therapeutics for the treatment of perivascular epithelioid cell tumors (PEComa) |
1 |
| Updates and challenges on treatment with BRAF/MEK-inhibitors in melanoma |
1 |
| Emerging treatment options for cholangiocarcinoma |
1 |
| Omics-driven drug repurposing as a source of innovative therapies in rare cancers |
1 |
| Cervical dystonia: an update on therapeutics |
1 |
| An overview of the efficacy and safety of deferiprone in paediatric patients with congenital haemoglobinopathies and chronic iron overload |
1 |
| Orphan medicinal products' access to the Bulgarian pharmaceutical market - challenges and obstacles |
1 |
| Advances in diagnosis and potential therapeutic options for familial chylomicronemia syndrome |
1 |
| Emerging and potential treatment options for sarcoidosis |
1 |
| Recent developments with defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome |
1 |
| Treatment options and dietary supplements for patients with phenylketonuria |
1 |
| Uridine triacetate-an antidote in the treatment of 5-fluorouracil or capecitabine poisoning |
1 |
| Sanfilippo type C disease: pathogenic mechanism and potential therapeutic applications |
1 |
| Advances in emerging therapeutics for oculopharyngeal muscular dystrophy |
1 |
| The dark matter of diffuse intrinsic pontine gliomas: an update |
1 |
| Development and validation of an MCDA framework for evaluation and decision-making of orphan drugs in Spain |
1 |
| Current biochemical treatments of mitochondrial respiratory chain disorders |
1 |
| The potential and benefits of repurposing existing drugs to treat rare muscular dystrophies |
1 |
| Alpha lipoic acid as a novel therapeutic approach to cystinuria |
1 |
| Advances on potential therapeutic options for epidermolysis bullosa |
1 |
| The clinical and molecular spectrum of autosomal dominant limb-girdle muscular dystrophies focusing on transportinopathy |
1 |
| The senseless orphanage of Chagas disease |
1 |
| Current management strategies and the potential of inhaled GM-CSF for the treatment of autoimmune pulmonary alveolar proteinosis |
1 |
| An update on treatment of ulcerative colitis |
0 |
| Progress and prospects for treating ataxia telangiectasia |
0 |
| Novel drug targets in idiopathic pulmonary fibrosis |
0 |
| Sydenham's chorea: an update on pathophysiology, clinical features and management |
0 |
| Alpha 1 antitrypsin deficiency: a rare multisystem disease, predominantly affecting the lung |
0 |
| Emerging treatments and therapeutic targets for tuberous sclerosis complex in children |
0 |
| Duodenal adenocarcinoma: neoadjuvant and adjuvant therapy strategies |
0 |
| Autoimmune hemolytic anemia - progress in emerging treatment options |
0 |
| Brigatinib for treatment of anaplastic lymphoma kinase-rearranged metastatic non-small cell lung cancer |
0 |
