Content about Genetic disorders

September 14, 2012
Lundbeck adds Omnicare to limited distribution network for Huntington's drug

The specialty pharmacy operated by Omnicare Specialty Care Group has been added to the limited distribution network for a drug used to treat a hereditary degenerative disorder.

ORLANDO, Fla. — The specialty pharmacy operated by Omnicare Specialty Care Group has been added to the limited distribution network for a drug used to treat a hereditary degenerative disorder.

Omnicare said Friday that Lundbeck had added Advanced Care Scripts to its network for Xenaziner (tetrabenazine). The drug is used to treat involuntary movements known as chorea associated with Huntington's disease. Chroea affects about 90% of people with Huntington's and is considered a hallmark of the disease, according to studies.

August 20, 2012
Rexall's annual vendor golf classic raises funds for Rexall Foundation

MISSISSAUGA, Ontario — Through the support of Rexall’s vendors and partners, Rexall’s annual vendor golf classic raised more than Canadian $250,000 for the Rexall Foundation earlier this month.

The Foundation for Prader-Willi Research received a C$10,000 donation at the Aug. 14 event.  The Foundation for Prader-Willi Research is an organization dedicated to eliminating the challenges of Prader-Willi Syndrome, a rare genetic disorder, through the advancement of research.

August 2, 2012
Safeway encourages customers to support MDA's 'Make a Muscle, Make a Difference' program

Safeway has commenced its annual fundraising campaign for the Muscular Dystrophy Association.

TUCSON, Ariz., and PLEASANTON, Calif. — Safeway has commenced its annual fundraising campaign for the Muscular Dystrophy Association.

Running through Sept. 3, the nationwide campaign gives customers the opportunity to contribute funds to MDA's "Make a Muscle, Make a Difference" program. Along with Safeway, Vons, Randalls, Tom Thumb, Dominick's and Carrs stores also will participate. Through its longstanding partnership, Safeway has raised more than $68.7 million for the MDA.

July 9, 2012
BioRx acquires Minn. bleeding-disorder pharmacy

Specialty pharmacy provider BioRx has acquired another specialty pharmacy provider based in Minnesota, BioRx said Monday.

CINCINNATI — Specialty pharmacy provider BioRx has acquired another specialty pharmacy provider based in Minnesota, BioRx said Monday.

BioRx, based in Cincinnati and specializing in such bleeding disorders as hemophilia, announced the purchase of Savage, Minn.-based Coagulife, which provides similar services.

June 26, 2012
Crohn's, colitis group launches redesigned website

An organization focused on Crohn's disease and ulcerative colitis has revamped its website, the group said Tuesday.

NEW YORK — An organization focused on Crohn's disease and ulcerative colitis has revamped its website, the group said Tuesday.

The Crohn's and Colitis Foundation of America said it worked with Pittsburgh-based digital marketing agency BarkleyREI to redesign the website. The new site includes a section for people newly diagnosed with the diseases; a library that includes webcasts, brochures, fact sheets and videos; social media integration; pages listing local chapters and support groups; and other resources.

May 29, 2012
BioPlus promotes Shelley Moore to lead bleeding disorders program

BioPlus Specialty Pharmacy has appointed a new person to head its bleeding disorders program, the company said Tuesday.

ALTAMONTE SPRINGS, Fla. — BioPlus Specialty Pharmacy has appointed a new person to head its bleeding disorders program, the company said Tuesday.

BioPlus announced the promotion of Shelley Moore to the position of national director of hemophilia. Moore already has served as head of the company's bleeding disorders program for Tennessee and has six years' nursing experience and a career specialty in bleeding disorders.

May 3, 2012
Orphan drugs to see growth through 2018, report finds

Drug makers could strike big with new treatments for rare diseases, according to a new report by a market research firm.

NEW YORK — Drug makers could strike big with new treatments for rare diseases, according to a new report by a market research firm.

May 2, 2012
FDA approves Pfizer, Protalix drug for Gaucher disease

The Food and Drug Administration has approved a new treatment for a rare genetic disorder that affects 10,000 people worldwide.

NEW YORK — The Food and Drug Administration has approved a new treatment for a rare genetic disorder that affects 10,000 people worldwide.

Pfizer and Protalix BioTherapeutics announced Wednesday the approval of Elelyso (taliglucerase alfa), an enzyme-replacement therapy for the long-term treatment of Type 1 Gaucher disease.

April 19, 2012
Hidden Valley to tout gluten-free promise on packaging

Hidden Valley is updating the packaging on its salad dressing products to address gluten-sensitive and gluten-intolerant customers.

OAKLAND, Calif. — Hidden Valley is updating the packaging on its salad dressing products to address gluten-sensitive and gluten-intolerant customers.

The company said it now will include a "gluten-free" logo to bottles and dry packets of its Original Ranch dressing, as well as much of its Farmhouse Originals product line. Packages with the new logo are slated to appear on store shelves this summer.

March 27, 2012
Hyperion buys rights to drug for rare genetic diseases

Hyperion Therapeutics has purchased rights to a drug for treating genetic metabolic disorders, the company said.

SOUTH SAN FRANCISCO, Calif. — Hyperion Therapeutics has purchased rights to a drug for treating genetic metabolic disorders, the company said.

Hyperion announced the purchase of Ravicti (glycerol phenylbutyrate) from Ucyclyd Pharma, a subsidiary of Medicis Pharmaceutical.

February 23, 2012
Despite gluten-free product boom, celiac disease may be widely undiagnosed

New research from Mintel suggested that the number of consumers that are gluten-intolerant/sensitive or have been diagnosed with celiac disease should be much greater.

CHICAGO — New research from Mintel suggested that the number of consumers that are gluten-intolerant/sensitive or have been diagnosed with celiac disease should be much greater.

February 16, 2012
More diagnoses to drive celiac disease market growth through decade

The global market for celiac disease is expected to grow substantially over the decade as more people are diagnosed with the condition, according to a new report.

LONDON — The global market for celiac disease is expected to grow substantially over the decade as more people are diagnosed with the condition, according to a new report.

The report, by British market research firm GlobalData, found that the global celiac disease therapeutics market would be worth more than $512 million by 2017 and $664.4 million by 2019.

January 12, 2012
Octapharma USA hires former Baxter BioScience executive

Drug maker Octapharma USA has appointed David Holliday as VP commercial development, the company said.

HOBOKEN, N.J. — Drug maker Octapharma USA has appointed David Holliday as VP commercial development, the company said.

January 5, 2012
Biogen Idec, Isis to develop spinal muscular atrophy drug

Biogen Idec and Isis Pharmaceuticals will work together on a drug invented by Isis for spinal muscular atrophy, the two companies said.

WESTON, Mass. — Biogen Idec and Isis Pharmaceuticals will work together on a drug invented by Isis for spinal muscular atrophy, the two companies said.

According to the companies, SMA is the most common genetic cause of infant mortality and causes muscle atrophy and weakness, occurring in 1-in-10,000 births. While children with the condition appear normal at birth, symptoms can develop within a few months and significantly shorten their lifespan. The drug, ISIS-SMNRx, is designed to compensate for the underlying genetic defect that causes SMA.

November 23, 2011
Shire seeks approval to make Gaucher disease drug at new plant

British drug maker Shire is looking to make a biotech drug for a rare genetic disorder at a new manufacturing plant in Massachusetts.

LEXINGTON, Mass. — British drug maker Shire is looking to make a biotech drug for a rare genetic disorder at a new manufacturing plant in Massachusetts.

The company said Tuesday that it had filed with the Food and Drug Administration and the European Medicines Agency for approval to produce the Gaucher disease drug Vpriv (velaglucerase alfa) at its Lexington, Mass., plant. The company expects to win approval to make the drug there early next year.

October 14, 2011
FDA approves treatment for iron overload

The Food and Drug Adminsitration has approved a drug made by ApoPharma for a complication resulting from treatments for a genetic condition that causes anemia, the agency said Friday.

SILVER SPRING, Md. — The Food and Drug Adminsitration has approved a drug made by ApoPharma for a complication resulting from treatments for a genetic condition that causes anemia, the agency said Friday.

The FDA announced the approval of Ferriprox (deferiprone), used to treat patients with iron overload due to blood transfusions for thalassemia, a blood disorder. Patients with the disease have excess iron in their bodies because of the frequent blood transfusions they must receive that can lead to iron overload, a condition that can be fatal.

September 15, 2011
GSK, Amicus start phase-3 trial of Fabry disease drug

GlaxoSmithKline and Amicus Therapeutics have started a late-stage clinical trial for a drug to treat a rare genetic disorder, the companies said.

CRANBURY, N.J. — GlaxoSmithKline and Amicus Therapeutics have started a late-stage clinical trial for a drug to treat a rare genetic disorder, the companies said.

GSK and Amicus announced the start of a second phase-3 trial of Amigal (migalastat hydrochloride) for Fabry disease, a rare lysosomal storage disorder that affects between 5,000 and 10,000 people worldwide. The market for Fabry disease has long been dominated by Genzyme, a subsidiary of French drug maker Sanofi that makes the biologic drug Fabrazyme (agalsidase beta).

September 7, 2011
Safeway raises $10.4 million for Muscular Dystrophy Association

After ending its five-week fundraising campaign to benefit the Muscular Dystrophy Association's efforts to develop treatments and assist people coping with neuromuscular diseases, Safeway announced that its stores raised $10.4 million for the organization.

PLEASANTON, Calif. — After ending its five-week fundraising campaign to benefit the Muscular Dystrophy Association's efforts to develop treatments and assist people coping with neuromuscular diseases, Safeway announced that its stores raised $10.4 million for the organization.

The campaign, which ran from Aug. 1 through Labor Day, marked Safeway's 10th annual fundraising effort. Safeway has raised more than $58 million for muscular dystrophy charities.

August 2, 2011
Safeway commences 10th annual MDA campaign

Safeway announced its 10th annual campaign to benefit the Muscular Dystrophy Association's efforts to develop treatments and assist people coping with neuromuscular diseases.

PLEASANTON, Calif. — Safeway announced its 10th annual campaign to benefit the Muscular Dystrophy Association's efforts to develop treatments and assist people coping with neuromuscular diseases.

June 14, 2011
FDA OKs infant-specific dose of Creon

The Food and Drug Administration has approved a new dosage of a drug made by Abbott for treating cystic fibrosis in infants, the drug maker said Tuesday.

ABBOTT PARK, Ill. — The Food and Drug Administration has approved a new dosage of a drug made by Abbott for treating cystic fibrosis in infants, the drug maker said Tuesday.

Abbott announced the approval of an infant-specific dose of Creon (pancrelipase) delayed-released capsules to treat exocrine pancreatic insufficiency due to cystic fibrosis.

June 8, 2011
Edison's EPI-743 granted orphan drug designation

The Food and Drug Administration has granted orphan drug designation to an investigational treatment made by Edison Pharmaceuticals for rare diseases, Edison said Wednesday.

MOUNTAIN VIEW, Calif. — The Food and Drug Administration has granted orphan drug designation to an investigational treatment made by Edison Pharmaceuticals for rare diseases, Edison said Wednesday.

April 28, 2011
Bayer extends sponsorship agreement with World Federation of Hemophilia

Bayer HealthCare Pharmaceuticals and the World Federation of Hemophilia announced Thursday a three-year extension of Bayer’s sponsorship agreement with the WFH.

MONTVILLE, N.J. — Bayer HealthCare Pharmaceuticals and the World Federation of Hemophilia announced Thursday a three-year extension of Bayer’s sponsorship agreement with the WFH.

The agreement calls for Bayer to serve as the sole sponsor of the WFH’s International Hemophilia Training Centre program and provide support for other activities of the organiztion, such as its website and Congress. The program trains healthcare professionals in developing countries in diagnosis and management of hemophilia and related bleeding disorders.

April 18, 2011
Lilly receives complete response letter for liprotamase

The Food and Drug Administration declined to approve a drug made by Eli Lilly for treating a disease of the pancreas that often occurs in patients with cystic fibrosis, Lilly said.

INDIANAPOLIS — The Food and Drug Administration declined to approve a drug made by Eli Lilly for treating a disease of the pancreas that often occurs in patients with cystic fibrosis, Lilly said.

April 7, 2011
Pfizer, Zacharon enter collaboration

Pfizer is partnering with San Diego biotech company Zacharon Pharmaceuticals to develop treatments for rare genetic disorders Zacharon said Thursday.

SAN DIEGO — Pfizer is partnering with San Diego biotech company Zacharon Pharmaceuticals to develop treatments for rare genetic disorders Zacharon said Thursday.

Under the collaboration, which could garner Zacharon up to $210 million, the companies will discover and develop pharmaceutical drugs for treating lysosomal storage disorders, a class of illnesses that includes Fabry disease, Gaucher disease, Pompe disease and Tay-Sachs disease.

March 21, 2011
MedPro Rx announces Education Is Power scholarship program

Patients with bleeding disorders could be eligible for a scholarship from a specialty pharmacy provider that specializes in treating them.

RALEIGH, N.C. — Patients with bleeding disorders could be eligible for a scholarship from a specialty pharmacy provider that specializes in treating them.

MedPro Rx announced Sunday that patients with such disorders as hemophilia and von Willebrand disease attending college and active in their communities could be eligible for its Education Is Power scholarship program.