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Nplate (romiplostim) Phase III study in children with symptomatic immune thrombocytopenia.-Amgen

Amgen has announced that The Lancet published results from a Phase III randomized, double-blind, placebo-controlled study of Nplate (romiplostim) in...

Drug news

16/12/18

FDA approves sBLA for NPlate to treat pediatric patients one year of age and older with immune thrombocytopenia. Amgen.

Amgen announced that the FDA has approved the supplemental Biologics License Application (sBLA) for Nplate (romiplostim) for the treatment of...

Journal

30/06/17

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial.

Background: Spinal muscular atrophy (SMA) is a progressive motor neuron disease causing loss of motor function and reduced life expectancy, for which limited treatment is available. We investigated the safety and efficacy of...

Source: The Lancet Neurology

Journal

29/05/18

Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial.

Introduction: This phase III, double-blind, randomised, placebo-controlled trial (and extension phase) was designed to assess the efficacy and safety of velmanase alfa (VA) in alpha-mannosidosis (AM) patients.

Source: Journal of Inherited Metabolic Disease

Journal

01/01/18

Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (PATH): a randomised, double-blind, placebo-controlled, phase 3 trial.

Subcutaneous immunoglobulin (SCIg) is an alternative option for immunoglobulin delivery, but has not previously been investigated in a large trial of CIDP. The PATH study compared relapse rates in patients given SCIg versus placebo.

Source: The Lancet Neurology

Journal

31/05/18

Eltrombopag in immune thrombocytopenia: efficacy review and update on drug safety.

This review summarizes the experience reported using eltrombopag in ITP, paying attention to efficacy and safety.

Source: Therapeutic Advances in Drug Safety

Drug news

14/11/24

First patient dosed in Greater China in global registrational clinical trial of efgartigimod in primary immune thrombocytopenia.

Zai Lab Limited announced that the first patient with primary immune thrombocytopenia (ITP) was treated with efgartigimod in Greater China (mainland China, Hong Kong, Macau, and Taiwan) as part of the global registrational ADVANCE-SC Phase III study.

Journal

31/12/20

Nebulised ALX-0171 for respiratory syncytial virus lower respiratory tract infection in hospitalised children: a double-blind, randomised, placebo-controlled, phase 2b trial

Background: Respiratory syncytial virus (RSV) is the most common cause of severe lower respiratory tract infection, with a high global health burden. There are no effective treatments available. ALX-0171 is a novel trivalent Nanobody with antiviral properties against RSV.

Source: The Lancet Respiratory Medicine

Journal

31/08/18

Apomorphine subcutaneous infusion in patients with Parkinson's disease with persistent motor fluctuations (TOLEDO): a multicentre, double-blind, randomised, placebo-controlled trial.

We aimed to investigate the efficacy and safety of apomorphine infusion compared with placebo in patients with Parkinson's disease with persistent motor fluctuations despite optimised oral or transdermal treatment.

Source: The Lancet Neurology

Journal

01/01/14

Methylphenidate hydrochloride modified-release in adults with attention deficit hyperactivity disorder: a randomized double-blind placebo-controlled trial.

Treatment options for adults with attention deficit hyperactivity disorder (ADHD) are limited. The study was conducted to confirm the clinically effective and safe dose of methylphenidate hydrochloride...

Source: Advances in Therapy

Find disease awareness content and relevant supporting materials

Nplate (romiplostim) Phase III study in children with symptomatic immune thrombocytopenia.-Amgen

FDA approves sBLA for NPlate to treat pediatric patients one year of age and older with immune thrombocytopenia. Amgen.

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial.

Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial.

Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (PATH): a randomised, double-blind, placebo-controlled, phase 3 trial.

Eltrombopag in immune thrombocytopenia: efficacy review and update on drug safety.

First patient dosed in Greater China in global registrational clinical trial of efgartigimod in primary immune thrombocytopenia.

Nebulised ALX-0171 for respiratory syncytial virus lower respiratory tract infection in hospitalised children: a double-blind, randomised, placebo-controlled, phase 2b trial

Apomorphine subcutaneous infusion in patients with Parkinson's disease with persistent motor fluctuations (TOLEDO): a multicentre, double-blind, randomised, placebo-controlled trial.

Methylphenidate hydrochloride modified-release in adults with attention deficit hyperactivity disorder: a randomized double-blind placebo-controlled trial.