Sarepta Therapeutics Announces USPTO Decision in Patent Interference Case with BioMarin Pharmaceutical

– Importance of USPTO decision not ascertainable until determinations are rendered in two remaining composition interferences and planned appeal – CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sep. 30, 2015– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Patent Trial and Appeal Board (PTAB) of the United States Patent and Trademark Office (USPTO) has . . .

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Five Questions with Researcher Criss Hartzell

Criss Hartzell, professor of cell biology at Emory University School of Medicine in Atlanta, Ga., recently was awarded an MDA research grant to study the ANO5 gene. Mutations in the gene have been implicated in both type 2L limb-girdle muscular dystrophy (LGMD) and a subtype of Miyoshi distal muscular dystrophy (a type of distal muscular dystrophy, . . .

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BioMarin Receives Favorable Ruling in the Use of Exon 51 Antisense Oligonucleotides Patent Interference

Priority of U.S. Patent Claims Confirmed by U.S. Patent Trial and Appeal Board (PTAB) SAN RAFAEL, Calif., Sept. 29, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Patent Trial and Appeal Board (PTAB) issued a decision in favor of BioMarin’s claims to the use of exon 51 antisense oligonucleotides to treat . . .

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$2 Million in New MDA Grants Target DMD

By Laura Hagerty, Ph.D., MDA’s DMD Scientific Program Officer On September 7, 2015, MDA will rally alongside our Duchenne muscular dystrophy (DMD) community — kids, adults, families, caregivers and others — as we recognize World Duchenne Awareness Day. And as we move toward that day, there’s a palpable feeling of excitement in the air. For . . .

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FDA Approves Drug for Primary Hyperkalemic and Hypokalemic Periodic Paralysis

Statement from MDA Executive Vice President and Chief Medical and Scientific Officer Valerie A. Cwik, M.D.: “We couldn’t be more pleased for our families affected by hyperkalemic and hypokalemic periodic paralysis, as the first treatment for these diseases has been approved by the U.S. Food and Drug Administration (FDA) and is expected to be available in . . .

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MDA to Offer Genetic Testing for Limb-Girdle Muscular Dystrophy

MDA, Genzyme and Emory University team up to expand access to genetic testing for accurate diagnosis of limb-girdle muscular dystrophy CHICAGO, March 26, 2015 — The Muscular Dystrophy Association announced today that thanks to a grant from Genzyme, a Sanofi company, and in collaboration with Emory Genetics Laboratory, MDA clinics are now offering genetic testing . . .

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Statement: New MDA-Funded Genetic Therapy Technique Targets DMD

CHICAGO, Feb. 27, 2015 – Muscular Dystrophy Association research grantee Charles Gersbach, Assistant Professor of Biomedical Engineering at Duke University, recently announced a potentially game-changing advance in gene modification for boys and young men with Duchenne muscular dystrophy (DMD). The results were published Feb. 18 in Nature Communications, and Gersbach will discuss their implications at MDA’s . . .

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ReveraGen BioPharma Announces Start of Phase 1 Clinical Trial of VBP15 Dissociative Steroid Drug

Product holds potential for replacing glucocorticoids in Duchenne muscular dystrophy and other chronic inflammatory states SILVER SPRING, M.D./ CHICAGO – February 18, 2015 – ReveraGen BioPharma today announced the start of a Phase 1, first-in-human, clinical trial of a novel dissociative steroidal drug, VBP15, under development for Duchenne muscular dystrophy.  Recruitment in the trial is . . .

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