Rare Disease News and Research

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Imbruvica gets FDA approval for treatment of mantle cell lymphoma

Imbruvica gets FDA approval for treatment of mantle cell lymphoma

Promethera Biosciences completes patient enrolment for first clinical trial of HepaStem

Promethera Biosciences completes patient enrolment for first clinical trial of HepaStem

Research findings may lead to new treatments, approaches for controlling multiple sclerosis

Research findings may lead to new treatments, approaches for controlling multiple sclerosis

Musicians can develop saxophone lung from fungus

Musicians can develop saxophone lung from fungus

Patrys receives FDA Orphan Medicinal Product Designation for PAT-SM6

Patrys receives FDA Orphan Medicinal Product Designation for PAT-SM6

FDA grants Orphan Drug Designation to Advaxis' ADXS-HPV for HPV-associated head and neck cancer

FDA grants Orphan Drug Designation to Advaxis' ADXS-HPV for HPV-associated head and neck cancer

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Viropharma announces financial results for third quarter 2013

Viropharma announces financial results for third quarter 2013

Two related genes underlie development of two rare bone tumours in nearly 100% of patients

Two related genes underlie development of two rare bone tumours in nearly 100% of patients

Complete Care program improves outcomes for Kaiser Permanente patients

Complete Care program improves outcomes for Kaiser Permanente patients

Illumina announces launch of TruGenome Undiagnosed Disease Test

Illumina announces launch of TruGenome Undiagnosed Disease Test

Thomson Reuters adds Cortellis Clinical Trials Intelligence for drug discovery and development

Thomson Reuters adds Cortellis Clinical Trials Intelligence for drug discovery and development

Researchers identify novel gene mutation that causes common variable immunodeficiency

Researchers identify novel gene mutation that causes common variable immunodeficiency

Researchers test ATR-101 compound in clinical trial for adrenal cancer

Researchers test ATR-101 compound in clinical trial for adrenal cancer

Two institutions receive $25M grant from NIH to abet clinical and translational research

Two institutions receive $25M grant from NIH to abet clinical and translational research

Liposorber LA-15 System to treat pediatric patients with primary FSGS gets FDA approval

Liposorber LA-15 System to treat pediatric patients with primary FSGS gets FDA approval

Drugs often fail to bring acromegaly under control in routine clinical practice

Drugs often fail to bring acromegaly under control in routine clinical practice

Mechanism preserving genome integrity help develop new therapies against DiGeorge syndrome

Mechanism preserving genome integrity help develop new therapies against DiGeorge syndrome

Lone atrial fibrillation in children has substantial symptomatic burden, recurrence rate

Lone atrial fibrillation in children has substantial symptomatic burden, recurrence rate

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