The Kerala High Court has allowed the mother of a three-year-old boy suffering from Spinal Muscular Atrophy (SMA) to buy Risdiplam, an imported drug for treatment of the rare disease, without...
The US Food and Drug Administration (FDA) approval in December 2023, of two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of a rare...
KOTTAYAM : Smitha Antony and Manu Joseph seem to have hit a brick wall in terms of their physical and emotional agony. The couple from Kozhuvanal, in Kottayam district, are...
A two-month-old baby girl in Mumbai has become one of the youngest patients to receive a bone marrow transplant from an unrelated donor. The baby was diagnosed with a rare...
Following Sanofi’s decision to terminate its proposed acquisition of an exclusive license to Maze Therapeutics Inc.’s developmental drug to treat Pompe disease, the Federal Trade Commission moved to dismiss its...
Pompe disease, a lysosomal storage disorder, is characterized by an impairment of glycogen degradation that is caused by a deficiency of the enzyme acid α-glucosidase (GAA).1 In efforts to expand the...
“I watch my 16-month-old daughter Sera bravely fight an ultra-rare disease every day,” said a tearful Michael Andrew, a desperate father waging a war not against the world but against...
Alexion, AstraZeneca Rare Disease, has completed a definitive purchase and license agreement for a portfolio of preclinical rare disease gene therapy programes and enabling technologies from Pfizer Inc. (Pfizer). These...
Our Executive Director Mr. Prasanna Shirol was a part of First Ever summit dedicated to advancing equitable care for rare diseases in older adults. The meeting was attended from 25 countries across Canada, Latin America, Europe, and Asia, representing patient and caregiver communities, patient advocates, healthcare professionals, and healthcare policy experts. This is a short video highlighting the impact of the initiative to date, in the words of the COLLABORATE Community.
