August 14, 2015

Keveyis is the first medicine approved by the FDA for the treatment of primary periodic paralysis, which is estimated to affect approximately 5,000 people in the United States.

August 10, 2015

In order to ensure that patients receive treatment for periodic paralysis, Taro will be partnering with Diplomat Pharmacy, where patients and caregivers will receive treatment access and support. 

August 3, 2015

The ALS Ice Bucket Challenge will return on Friday.

May 29, 2015

The Food and Drug Administration Friday announced that it had approved Rapamune (sirolimus), distributed by Pfizer’s Wyeth Pharmaceuticals, as a treatment for the rare lung disease lymphangioleiomyomatosis (LAM). 

March 13, 2015

The Tuberous Sclerosis Alliance will honor Helena Foulkes, president of CVS/pharmacy and EVP of CVS Health, at the organization's 14th Annual Comedy for a Cure in April.

August 20, 2014

The Food and Drug Administration has approved Cerdelga (eliglustat) for the treatment of adult patients with the Type 1 form of Gaucher disease.

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.