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New Data Validate Amicus’s Pompe Disease Candidate

New Data Validate Amicus's Pompe Disease Candidate

Summary Amicus announced additional positive data from its phase 1/2 study in Pompe disease. The data confirm the potential of ATB200/AT2221 to become the new standard of care in Pompe disease. Increasing my price target to $17 based due to the addition of the Pompe combo to my model. Amicus Therapeutics (NASDAQ:FOLD) reported additional positive […]

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Pompe Disease: A Multidisciplinary Approach

Pompe Disease: A Multidisciplinary Approach

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February 2017 Update from IPA

February 2017 Update from IPA

  Click here to download the pdf version.

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Gene Therapy Effective For Pompe Disease In Mice Prepared For Human Trials: Study

Gene Therapy Effective For Pompe Disease In Mice Prepared For Human Trials: Study

Duke Health researchers have developed a gene therapy that could enhance or even replace the only FDA-approved treatment currently available to patients with a rare, life-threatening condition that cripples the muscles. The condition known as Pompe disease is inheritable and affects around 1 in every 20,000 babies and can also affect adults. The gene therapy […]

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September 2016 Update from IPA

September 2016 Update from IPA

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For genome services, India must be put on the map

Prajwal Hulluraiah is engrossed in a game on his mother’s mobile phone and wouldn’t look up to talk to you. But that’s where his similarity with any other 11-year-old ends. Twice a month, Prajwal checks into the Indira Gandhi Institute of Child Health (IGICH) in Bangalore for a six-hour procedure to replace the enzymes in […]

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Gene therapy used to treat pulmonary dysfunction in Pompe disease

The team at UF’s Powell Center for Rare Disease Research and Therapy conducted the first in-human study of gene therapy to treat respiratory dysfunction in patients with infantile onset Pompe. In the study, which began in 2011, an adeno-associated virus vector was used to carry a functional copy of the affected gene to muscle cells […]

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Precision Medicine Can Offer Hope to Rare Disease Patients

Precision Medicine Can Offer Hope to Rare Disease Patients

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A father’s mission: How one man developed a drug to save the lives of his children

In 1996, John Crowley’s two youngest children received what doctors at the time believed to be a death sentence. John and his wife, Aileen, had noticed that their daughter, Megan, who was 15 months old at the time, had not yet taken her first steps and wasn’t pulling up in her crib. “We got Megan’s […]

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Uncertainty for Masterton mother after drug trial ditched

Uncertainty for Masterton mother after drug trial ditched

In a few weeks’ time, Allyson Lock’s fate will be left “swinging in the wind”. The Masterton mother of three has Pompe disease, a rare inherited degenerative disorder which is almost always fatal. For the past five years Mrs Lock, 51, has been on a drug trial run by pharmaceutical giant BioMarin. “The drug was […]

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