Myasthenic Syndrome News and Research

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A bitter battle over the ‘orphan drug’ program leaves patients’ pocketbooks at risk

A bitter battle over the ‘orphan drug’ program leaves patients’ pocketbooks at risk

Researchers investigate a drug used for neuromuscular weakness as potential treatment for botulism

Researchers investigate a drug used for neuromuscular weakness as potential treatment for botulism

New research shines light on mysterious dynamics that define the interplay between nerves and muscle

New research shines light on mysterious dynamics that define the interplay between nerves and muscle

FDA approves new drug for treatment of rare autoimmune disorder

FDA approves new drug for treatment of rare autoimmune disorder

Combination of Opdivo and Yervoy shows four-year survival benefits in patients with advanced melanoma

Combination of Opdivo and Yervoy shows four-year survival benefits in patients with advanced melanoma

Opdivo plus low-dose Yervoy shows promising results in treating renal cell carcinoma patients

Opdivo plus low-dose Yervoy shows promising results in treating renal cell carcinoma patients

Study identifies potential genetic link in sudden infant death syndrome

Study identifies potential genetic link in sudden infant death syndrome

Research provides new insight into how diseases that disconnect brain and body occur

Research provides new insight into how diseases that disconnect brain and body occur

Amgen and Merck expand collaboration for Talimogene laherparepvec-KEYTRUDA combination study

Amgen and Merck expand collaboration for Talimogene laherparepvec-KEYTRUDA combination study

Intravenous immune globulin effective treatment for GBS, CIDP

Intravenous immune globulin effective treatment for GBS, CIDP

BioMarin to initiate BMN-111 Phase 1 trial in achondroplasia

BioMarin to initiate BMN-111 Phase 1 trial in achondroplasia

EMA CHMP recommends approval for BioMarin's Novato manufacturing facility expansion

EMA CHMP recommends approval for BioMarin's Novato manufacturing facility expansion

BioMarin commences GALNS Phase 2 study in mucopolysaccharidosis IVA

BioMarin commences GALNS Phase 2 study in mucopolysaccharidosis IVA

Positive data from BioMarin's MOR-100 Phase 1/2 extension study on Morquio syndrome

Positive data from BioMarin's MOR-100 Phase 1/2 extension study on Morquio syndrome

BioMarin commences BMN 673 Phase 1 trial in hematological malignancies

BioMarin commences BMN 673 Phase 1 trial in hematological malignancies

BioMarin to acquire Pfizer's Ireland manufacturing plant

BioMarin to acquire Pfizer's Ireland manufacturing plant

BioMarin announces preliminary results from ADAPT in patients suffering from PKU

BioMarin announces preliminary results from ADAPT in patients suffering from PKU

Gene GFPT1 crucial in causing variation of Congenital Myasthenic Syndrome

Gene GFPT1 crucial in causing variation of Congenital Myasthenic Syndrome

BioMarin commences GALNS Phase 3 trial in patients with Morquio A Syndrome

BioMarin commences GALNS Phase 3 trial in patients with Morquio A Syndrome

Biomarin commences BMN 701 Phase 1/2 trial for treatment of Pompe disease

Biomarin commences BMN 701 Phase 1/2 trial for treatment of Pompe disease

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