Myasthenic Syndrome News and Research

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

A prescription drug that helps Lore Wilkinson walk and talk despite a rare muscle disease cost her so little for more than a decade that she didn't even use her insurance to pay for it.

22 Feb 2023

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

Wake Forest Institute for Regenerative Medicine (WFIRM) researchers are investigating a drug used to treat neuromuscular weakness as a potential treatment for botulism, a rare but serious disease.

25 Jul 2022

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

New research co-led by a University of Ottawa Faculty of Medicine professor is shedding light on some of the mysterious dynamics that define the interplay between nerves and muscle.

17 Feb 2022

FDA approves new drug for treatment of rare autoimmune disorder

The U.S. Food and Drug Administration today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults.

29 Nov 2018

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

Bristol-Myers Squibb Company today announced four-year data from the Phase 3 CheckMate -067 clinical trial - the longest follow-up to date - which continues to demonstrate durable, long-term survival benefits with the first-line combination of Opdivo (nivolumab) and Yervoy (ipilimumab), versus Yervoy alone, in patients with advanced melanoma.

22 Oct 2018

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

Bristol-Myers Squibb Company today announced patient-reported outcomes data from the Phase 3 CheckMate -214 trial in intermediate- and poor-risk patients with advanced renal cell carcinoma treated with the Immuno-Oncology combination Opdivo plus low-dose Yervoy versus sunitinib over a two-year follow-up period.

1 Jun 2018

Study identifies potential genetic link in sudden infant death syndrome

Rare genetic mutations associated with impairment of the breathing muscles are more common in children who have died from sudden infant death syndrome (also known as ‘cot death’) than in healthy controls, suggesting a possible genetic element of the disorder, according to a case-control study in the UK and USA published in The Lancet.

29 Mar 2018

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

A huge colony of receptors must be optimally positioned and functioning on our muscle cells for our brains to talk with our bodies so we can walk and breathe.

29 Nov 2016

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

Amgen and Merck, known as MSD outside the U.S. and Canada, today announced an expanded collaboration to evaluate the efficacy and safety of talimogene laherparepvec, Amgen's investigational oncolytic immunotherapy, in combination with KEYTRUDA® (pembrolizumab), Merck's anti-PD-1 therapy, in a Phase 1, open-label trial of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN).

1 Jun 2015

Intravenous immune globulin effective treatment for GBS, CIDP

Intravenous immune globulin (IVIg) is an effective treatment for certain disorders of the nerve and muscles, including Guillain-Barré syndrome (GBS) and a form of neuropathy called chronic inflammatory demyelinating polyneuropathy (CIDP), according to a guideline issued by the American Academy of Neurology.

27 Mar 2012

BioMarin to initiate BMN-111 Phase 1 trial in achondroplasia

BioMarin Pharmaceutical Inc. announced today that the Investigational New Drug (IND) application for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia is active.

4 Jan 2012

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

BioMarin Pharmaceutical Inc. announced today that the Committee for Medicinal Products for Human Use of the European Medicines Agency has recommended approval for the company's manufacturing facility expansion in Novato, CA.

19 Dec 2011

BioMarin commences GALNS Phase 2 study in mucopolysaccharidosis IVA

BioMarin Pharmaceutical Inc. today announced the initiation of a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA.

30 Nov 2011

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

BioMarin Pharmaceutical Inc. today announced an update on the GALNS Phase 1/2 extension study (MOR-100) in which patients have continued treatment on an ongoing basis.

29 Jul 2011

BioMarin commences BMN 673 Phase 1 trial in hematological malignancies

BioMarin Pharmaceutical Inc. today announced the initiation of a Phase 1 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, for the treatment of patients with advanced hematological malignancies.

15 Jul 2011

BioMarin to acquire Pfizer's Ireland manufacturing plant

BioMarin Pharmaceutical Inc. announced today that it has entered into a definitive agreement to acquire a bulk biologics manufacturing plant from Pfizer, located in Shanbally, Cork, Ireland.

24 Jun 2011

BioMarin announces preliminary results from ADAPT in patients suffering from PKU

BioMarin Pharmaceutical Inc. announced today preliminary results from ADAPT (A Diversified Approach for PKU Treatment) in an abstract (Abstract #91, Mental Health Screening in Phenylketoniuria Clinic) presented by Barbara Burton, MD at the 2011 annual American College of Medical Genetics conference in Vancouver, Canada.

19 Mar 2011