August 4, 2014

The Food and Drug Administration approved Lumizyme (alglucosidase alfa) for treatment of patients with infantile-onset Pompe disease, including patients who are younger than 8 years of age.

February 18, 2014

The Food and Drug Administration has approved Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome), which is a rare disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS).

October 31, 2013

Earlier this year, the Food and Drug Administration approved a new drug for treating cystic fibrosis for patients who have a specific genetic mutation, but that also treats the underlying cause of the disease rather than the disease itself.

July 31, 2013

Nearly 450 drugs are under development for treating neurological disorders, according to a report by a drug industry trade group.

June 11, 2013

Aceto Corp. will sell an authorized generic version of a drug used to treat a rare but devastating degenerative disorder.

June 3, 2013

CVS/pharmacy has announced the launch of its annual in-store Advancing Medical Research fundraising campaign to support medical research and help improve the quality of life for those living with amyotrophic lateral sclerosis (ALS).

May 10, 2013

A biotech company and a non-profit organization are looking into new ways to treat patients with the devastating degenerative disorder commonly known as Lou Gehrig's disease.

January 3, 2013

An experimental drug for Lou Gehrig's disease made by Biogen Idec has failed in a late-stage clinical trial, the drug maker said.

November 19, 2012

Drug maker Shire has started a research program focused on rare diseases and their effects on patients and healthcare professionals, the company said Monday.

November 1, 2012

The global market for treatments for neurodegenerative disorders will exceed $20 billion before the end of the decade, according to a new report.

October 2, 2012

An experimental drug for treating a rare genetic disease appears effective, according to a late-stage clinical trial.

September 10, 2012

Amyotrophic lateral sclerosis patients from Chagrin Falls, Ohio, and cystic fibrosis patients from Indianapolis took part in pre-game, on-field traditions at Fenway Park to celebration CVS/pharmacy's Advancing Medical Research fundraising campaign, which raised more than $5 million in five weeks.

August 29, 2012

In the battle to find a cure for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, scientists from Belgium have made a rare discovery: a new gene that influences survival time of ALS. The findings, reported in a recent issue of Nature Medicine, were funded in part by CVS/pharmacy and the ALS Therapy Alliance, a Boston-based nonprofit.

May 29, 2012

CVS/pharmacy has announced the launch of a new in-store fundraising campaign, named Advancing Medical Research, to support medical research and help improve the quality of life for those living with amyotrophic lateral sclerosis and cystic fibrosis.

May 3, 2012

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

May 2, 2012

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

November 23, 2011

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

September 1, 2011

Country music star Ty Herndon and former NFL player Kevin Turner joined amyotrophic lateral sclerosis patients Thursday evening at Fenway Park to celebrate the success of CVS/pharmacy’s annual in-store fundraising campaign to support the fight against ALS.

June 6, 2011

CVS/pharmacy kicked off on June 5 its 10th annual in-store "Researching a Cure" fundraising campaign to support the fight against amyotrophic lateral sclerosis.

April 18, 2011

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

February 16, 2011

French drug maker Sanofi-Aventis will acquire U.S. biotech giant Genzyme for $20.1 billion, under an agreement the two companies announced Wednesday.

January 10, 2011

It appears that French drug maker Sanofi-Aventis’ efforts to acquire U.S. biotech company Genzyme are getting somewhere, according to company and media reports.

December 13, 2010

Biotech company Genzyme still is saying “no thanks” to French drug maker Sanofi-Aventis’ tender offer of $18.5 billion, or $69 per share, to acquire it, saying the offer “substantially undervalues” the company.