Patient management and therapy , 

Alkaptonuria: A review of the potential of nitisinone for the treatment of alkaptonuria

• Consult the abstract

• To read more about “Alkaptonuria”

• Expert Opinion on Orphan Drugs, 15 Sep 2019 : 1-7

Hepatic veno-occlusive disease: A review of recent developments with defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome

• Consult the abstract

• To read more about “Hepatic veno-occlusive disease”

• Expert Opinion on Orphan Drugs 2019, Vol.7, Nos.7-8, 337-347

Spinal muscular atrophy: A review of a novel modality in gene therapy, AAV9 vector

• Consult the pubmed abstract

• To read more about “Proximal spinal muscular atrophy”
• To read more about “Distal hereditary motor neuropathy”

• Gene Ther. 2019 Aug;26(7-8):287-295

Marfan syndrome: A review of the pathophysiology, treatment and genetics

• Consult the abstract

• To read more about “Marfan syndrome”

• Expert Opinion on Orphan Drugs Vol. 4, 2016, No.6, 605-612

Cystic fibrosis: A review of the perspective on gene therapy and small molecule treatment beyond cystic fibrosis transmembrane conductance regulator therapy

• Consult the pubmed abstract

• To read more about “Cystic fibrosis”

• Gene Ther. 2019 Sep;26(9):354-362

Tuberous sclerosis complex: A review of emerging treatments and therapeutic targets in children

• Consult the abstract

• To read more about “Tuberous sclerosis complex”

• Expert Opinion on Orphan Drugs Vol. 7, No.9, 373-381

Hereditary retinal degenerations: Improvement in vision with gene therapy and oral retinoid administration

• Consult the pubmed abstract

• To read more about “Isolated inherited retinal disorder”

• Expert Opin Orphan Drugs. 2015 May 4;3(5):563-575

Fibrogenesis imperfecta ossium : A review of clinical approach to diagnosis and management of a rare skeletal disorder

• Consult the pubmed abstract

• Expert Opin Orphan Drugs. 2015 May 4;3(5):563-575

Adult medulloblastoma: An overview on current and future strategies of treatment

• Consult the pubmed abstract

• To read more about “Medulloblastoma”

• Expert Opinion on Orphan Drugs 2019, Vol.7, No.9 : 383-389